• Welcome to Global Perspectives #3 2024

    HAEi’s CEO and Chairman of the Board, Anthony J. Castaldo, and HAEi’s President, Henrik Balle Boysen

    Dear HAEi Friends,

    We welcome you to this third issue of Global Perspectives with great optimism for the future, energized by the trailblazing collaboration, determination, motivation, and shared purpose on display at the 2024 HAEi Global Leadership Workshop (GLW) and the ACARE Global Angioedema Forum 2024 (GAF). Total attendance exceeded 750 people representing 90 countries and included patient leaders, healthcare professionals, and industry representatives. As you will read in this magazine, remarkable and results-oriented, these events ran simultaneously and culminated in an exciting half-day joint session where “GAF Meets GLW.”

    This issue also highlights the incredible HAE advocacy activities taking place in every corner of the globe—from the advancements in the Asia-Pacific region as we gear up for the 2025 Regional Conference APAC in Manila to the inspiring progress made in Sub-Saharan Africa, Central America, and Europe. The appointment of new Regional Patient Advocates and the realignment of responsibilities within our team have strengthened our ability to provide tailored support to Member Organizations (MO) in every region of the globe.

    HAEi’s heart and soul revolve around our steadfast commitment to helping our MOs flourish by providing the tools needed to fulfill their mission. In the article about GLW and GAF, you will read about transformative resources like the HAEi FocalPoint portal, which enhances communication between our MOs and HAEi, and the upcoming release of the next version of the HAE TrackR app, designed to give patients even more control over managing and documenting their HAE. We also spotlight the continued development of educational modules in the HAEi Advocacy Academy that offer training to MOs and the next generation of HAE advocacy leaders.

    Looking ahead to 2025, we are excited about the new class of HAEi LEAP students who will be learning the project management, communications, and leadership skills required to ensure the success of future HAE advocates. We also look forward to accelerating ongoing advocacy efforts through the previously mentioned 2025 HAEi Regional Conference APAC that will take place in Manila. This conference, which will revolve around the theme, “Embrace Opportunities,” reflects our enduring commitment to providing HAEi friends in each region with opportunities to share experiences, expand knowledge, strengthen advocacy skills, and connect with healthcare professionals and industry representatives.

    The HAEi Board of Directors and the entire HAEi Team are continuously inspired by the unwavering dedication and meaningful progress being accomplished by HAEi friends throughout the world. Your efforts have a significant, positive impact, and your contributions inspire even more concerted action to create a world where everyone affected by HAE can live a full, healthy, and empowered life.

    With this issue, Global Perspectives magazine takes an exciting leap forward by transitioning to an online format, offering readers the convenience of accessing the magazine in their preferred language* with just a few clicks. This evolution not only enhances accessibility but also ensures that a wider global audience can stay informed about the latest developments in HAE care, advocacy, and research. By breaking down language barriers and embracing digital innovation, we aim to connect and empower even more individuals within our community. For those who prefer the traditional experience, the Magazine PDF will still be available, ensuring everyone can enjoy the content in the format they prefer.

    We hope this issue of Global Perspectives serves as a source of inspiration for being an active part of the global HAE movement, which aims to create a brighter future for people with HAE and their families.

    Anthony J. Castaldo


    Chief Executive Officer and Chairman of the Board, HAE International (HAEi)

    Henrik Balle Boysen


    President, HAE International (HAEi)

Update from HAEi’s Global Advocacy and Operations

  • Fiona Wardman, Executive Vice President Global Advocacy and Chief Diversity Officer

    Update from Global Advocacy

    The success of the recent 2024 HAEi Global Leadership Workshop and ACARE Global Angioedema Forum 2024 stands as a testament to the global movement HAEi has created for hereditary angioedema (HAE) advocacy. With participation from 90 member countries and 750 attendees representing patients, healthcare professionals, and industry partners, these gatherings were not just events but milestones. We know that the collective power of global patient advocacy can improve the lives of those affected by HAE.

    Strengthening our global outreach and support
    To further enhance our on-the-ground outreach and support, we welcome the appointment of a new Regional Patient Advocate (RPA). The latest addition to the team provided the opportunity to reallocate some responsibilities to ensure every region receives the targeted attention it needs. The changes will help HAEi reach more patients and Member Organizations (MO) while fostering more opportunities for collaboration and growth.

    Our new RPA, Fanny Schappler, will support countries in the Asia-Pacific (APAC) region, amongst others. The APAC region focus is particularly exciting, as it coincides with our upcoming 2025 HAEi Regional Conference APAC in Manila, Philippines. Building on the success of the 2023 HAEi Regional Conference APAC, next year’s meeting promises to offer an even better platform for patients, caregivers, physicians, and industry partners to come together. We will build on the momentum generated in 2023 and create more opportunities for awareness, advocacy, and access to modern treatments across the region.

    The 2024 HAEi Regional Conference Americas in Panama was a resounding success, showcasing the tireless dedication of our community. My heartfelt thanks go to everyone who participated and contributed to the event. Each HAEi conference is carefully tailored to the region it serves and fosters a deep sense of community among attendees. At each meeting, I am reminded of the incredible strength and passion within the HAEi network, and I eagerly anticipate the upcoming events in 2025.

    A global community dedicated to change
    HAEi continually expands its reach—locating more patients, supporting new MOs, and intensifying efforts to increase global awareness and education programs. HAEi’s resources and tools, available in more languages than ever before and tailored to the needs of our community, continue to evolve and improve. By working closely with pharmaceutical companies and MOs, we see improved access to modern therapies and more widespread reimbursement for HAE treatments in many countries.

    Reflecting on our collective journey, I am continually inspired by the dedication shown by our community. From the tireless efforts of individual MOs to the incredible work of the HAEi team, the commitment to improving the lives of those with HAE—both locally and globally—is truly commendable. With a shared vision and relentless drive, we will continue to build a future where every person with HAE has access to the care and support they need to live full and healthy lives.

  • Jørn Schultz-Boysen, Executive Vice President Global Operations and Chief Compliance Officer

    Update Global Operations

    It’s hard to believe how quickly time passes. While the recent 2024 HAEi Global Leadership Workshop (GLW) and ACARE Global Angioedema Forum 2024 (GAF) in Copenhagen are still fresh in our minds, I find myself reflecting on the lasting impact of the 2024 HAEi Regional Conference Americas, held in Panama earlier this year. Though some time has passed, the feedback from the post-conference survey confirms that the event continues to leave a lasting impression, offering valuable insights that help us shape and improve future gatherings for everyone involved.

    Building on the momentum from Panama, we recently gathered over 750 participants from 90 countries for the 2024 GLW and GAF held in Copenhagen in October. This joint event offered our global HAE community an unparalleled opportunity to come together, learn from one another, exchange ideas, and network. Bringing together key opinion leaders from all Member Organizations (MO), dedicated physicians, and representatives from the pharmaceutical industry presented a tremendous opportunity to further enhance and build on the quality of life for people with HAE. Thank you to everyone for participating in this highly impactful event.

    Also, a big thank you goes to our entire team for their extraordinary work in planning and executing such a significant event.

    I am excited to announce that a new release of our HAE TrackR app is just around the corner. The app’s improved features will continue to support patients in taking control of their HAE by offering them a better user experience and even more helpful tools.

    For our MOs, HAEi FocalPoint was launched just before the GLW in Copenhagen. This portal serves as a central hub for accessing and utilizing HAEi’s tools and resources, helping to keep communication flowing between MOs and HAEi, while enabling MOs to make the most of the support available to them.

    Looking forward to 2025, we are already planning for the 2025 HAEi Regional Conference APAC, which will be held in Manila, Philippines, from 7-9 March 2025. We are excited to bring together the HAE community from the Asia-Pacific region countries once again, with dedicated tracks for patients/caregivers, healthcare professionals, and youngsters.

    Lastly, the HAEi LEAP 2025 application process is now open, and we are eager to see all the exciting and innovative ideas that the next generation of young leaders and their MOs will bring to the table. We look forward to the creative and impactful projects resulting from this program.

    As always, I encourage you to explore haei.org to learn more about the tools, resources, and programs available to you. We look forward to continuing our shared mission of improving the lives of people living with HAE worldwide.

    This fall, Global Perspectives magazine evolves into an online format, allowing readers to access the magazine in their preferred language. This step makes the publication more accessible and allows many more people to stay current on the latest HAE care and advocacy developments. We’ll still have the Magazine PDF available if you want to read the news that way.

NEWS FROM HAEI’S REGIONAL PATIENT ADVOCATES

  • News from HAEi's Regional Patient Advocates

    News from Director, Regional Patient Advocate Program

    Michal Rutkowski, Director, Regional Patient Advocate Program

    HAEi Regional Patient Advocates – We help HAE people navigate the future!

    “Alone, we can do so little; together, we can do so much.”

    The above quote of the late and great Helen Keller, a world-famous speaker, author, and advocate for people with disabilities, refers to humans’ capability to achieve goals above and beyond their inventiveness if only we work together. Despite the passage of time, this quote remains relevant and even more significant nowadays.

    It should never be forgotten that “Patients with a deficiency of C1 inhibitor are not just interesting models for study, however; they are critically ill. Many have ancestors who died suddenly from asphyxia… Patients live in constant dread of life-threatening laryngeal obstruction.” These characteristics of people with HAE, written by the pioneers of HAE, Prof. Agostini and Prof. Cicardi, rightly describe the disease and its burden that has accompanied patients through generations.

    When HAEi was established in 2004, a promise was made to patients. Since then, HAEi has been fulfilling this promise to help everyone with HAE access and get reimbursement for modern treatment options. And it was back then when the world’s HAE community first united to work together.

    After 20 years of tireless work, 750 healthcare professionals, patient leaders, and industry representatives from 90 countries met for the 2024 HAEi Global Leadership Workshop and ACARE Global Angioedema Forum 2024. The largest-ever gathering to advance angioedema science, clinical care, and patient advocacy was the culmination of over two decades of efforts and activities to benefit people with HAE.

    I am delighted that at the center of the GLW were HAEi Regional Patient Advocates (RPA), whose contribution played a fundamental role in the meeting’s ultimate success. Regardless of the region the RPA covers or the language the RPA speaks, they serve HAEi Member Organizations (MO) and HAE patients with complete transparency, mutual understanding, and respect. Indeed, they are HAEi’s backbone and HAEi’s feet on the ground in the regions. To have this opportunity to collaborate with HAEi RPAs makes me proud of humanity in the 21st century as they are an example of how strong, compassionate, and humble personalities, modern technologies, and advanced science can be used to help and unite people.

    The RPAs were instrumental in delivering the breakout sessions at the GLW. You can read more about what went on in each session in the summaries of the RPA work. I’m also delighted to welcome our newest RPA, Fanny Schappler, who tells us more about herself in her first RPA report. Please take a moment to check all the summaries; there will be lots to capture your interest. If you are a newcomer, not yet connected with HAE peers, please contact one of our Regional Patient Advocates, who would be happy to assist you.

    I would like to end by quoting one of the greatest minds of the 20th century, Stephen Hawkins:

    “We are very, very small, but we are profoundly capable of very, very big things.”

    Given Prof. Hawkins’ scope of interest, the first part of the quote most likely refers to what a small part of the cosmos we are, but surely the next part indicates that humanity can achieve big things, and so can HAEi RPAs (in the right proportions, so to speak 😀).

    Stay tuned!

    Michal Rutkowski
    Director, Regional Patient Advocate Program

    Michal Rutkowski, Director, Regional Patient Advocate Program

    HAEi Regional Patient Advocates – We help HAE people navigate the future!

    “Alone, we can do so little; together, we can do so much.”

    The above quote of the late and great Helen Keller, a world-famous speaker, author, and advocate for people with disabilities, refers to humans’ capability to achieve goals above and beyond their inventiveness if only we work together. Despite the passage of time, this quote remains relevant and even more significant nowadays.

    It should never be forgotten that “Patients with a deficiency of C1 inhibitor are not just interesting models for study, however; they are critically ill. Many have ancestors who died suddenly from asphyxia… Patients live in constant dread of life-threatening laryngeal obstruction.” These characteristics of people with HAE, written by the pioneers of HAE, Prof. Agostini and Prof. Cicardi, rightly describe the disease and its burden that has accompanied patients through generations.

    When HAEi was established in 2004, a promise was made to patients. Since then, HAEi has been fulfilling this promise to help everyone with HAE access and get reimbursement for modern treatment options. And it was back then when the world’s HAE community first united to work together.

    After 20 years of tireless work, 750 healthcare professionals, patient leaders, and industry representatives from 90 countries met for the 2024 HAEi Global Leadership Workshop and ACARE Global Angioedema Forum 2024. The largest-ever gathering to advance angioedema science, clinical care, and patient advocacy was the culmination of over two decades of efforts and activities to benefit people with HAE.

    I am delighted that at the center of the GLW were HAEi Regional Patient Advocates (RPA), whose contribution played a fundamental role in the meeting’s ultimate success. Regardless of the region the RPA covers or the language the RPA speaks, they serve HAEi Member Organizations (MO) and HAE patients with complete transparency, mutual understanding, and respect. Indeed, they are HAEi’s backbone and HAEi’s feet on the ground in the regions. To have this opportunity to collaborate with HAEi RPAs makes me proud of humanity in the 21st century as they are an example of how strong, compassionate, and humble personalities, modern technologies, and advanced science can be used to help and unite people.

    The RPAs were instrumental in delivering the breakout sessions at the GLW. You can read more about what went on in each session in the summaries of the RPA work. I’m also delighted to welcome our newest RPA, Fanny Schappler, who tells us more about herself in her first RPA report. Please take a moment to check all the summaries; there will be lots to capture your interest. If you are a newcomer, not yet connected with HAE peers, please contact one of our Regional Patient Advocates, who would be happy to assist you.

    I would like to end by quoting one of the greatest minds of the 20th century, Stephen Hawkins:

    “We are very, very small, but we are profoundly capable of very, very big things.”

    Given Prof. Hawkins’ scope of interest, the first part of the quote most likely refers to what a small part of the cosmos we are, but surely the next part indicates that humanity can achieve big things, and so can HAEi RPAs (in the right proportions, so to speak 😀).

    Stay tuned!

    Michal Rutkowski
    Director, Regional Patient Advocate Program

  • News from North America and South East Asia

    From Regional Patient Advocates Anthony J. Castaldo and Henrik Balle Boysen

    Over the past several months, we have seen some very positive developments in the North America and Southeast Asia regions. Each of our countries brought a strong contingent of leaders to the 2024 HAEi Global Leadership Workshop, and we were delighted with the solid number of physicians from the region who attended the ACARE Global Angioedema Forum 2024.

    HAEi was honored to deliver a presentation on HAEi Advocacy Academy at an August HAE Canada (HAEC) Patient Information Update in Vancouver. We are also pleased to be in the process of working with HAEC to implement the HAEi Clinical Trial Awareness Framework for a trial that will soon begin recruiting in Canada. HAEC is a mature, experienced, and sophisticated organization, and we’re delighted that the Trial Awareness Framework package will serve as a useful supplement to their clinical trial awareness and recruitment efforts. Finally, we were pleased to engage in discussions with the HAEC President and provide HAEi’s perspectives and experience as the organization prepared for a meeting to update the Canadian/International HAE Guidelines.

    HAEi’s continued close collaboration with HAE Philippines (HAEP) is beginning to bear fruit! Together, we are working closely with three Allergy/Immunology specialists who have a special interest in educating clinicians on HAE symptoms, increasing the diagnosis rate, and advocating to bring modern treatments to the country. These physicians have already implemented our HAEi Heat Map survey and are following up on cases identified through the project. They have also requested that we work with them and HAEP to conduct our Baseline Burden of Illness Study.

    Throughout the summer months, HAEi, HAEP, and the active physicians have engaged in numerous discussions regarding strategies for taking action to engage with pharmaceutical companies and the Philippine government. We imparted our experience and provided examples of the bold steps and creative actions required to access modern medicines. We are very excited to report that our colleagues in the Philippines held successful meetings in September with a pharmaceutical company and a high-level official in the Department of Health. We are excited to report that (1) the pharmaceutical company has expressed interest in bringing medicine to the Philippines, and (2) the government representative disclosed there is funding for rare disease medications and pledged to work with the doctors and HAEP to bring desperately needed treatment into the Philippines.

    In addition, we are working with HAEP to:

    • add content to their website
    • make the ER poster available at key hospital centers
    • organize a patient meeting
    • broaden the use of HAEi Advocacy Academy, and
    • conduct an online seminar to introduce HAE TrackR.

    >> Meet the Regional Patient Advocates

  • News from East Asia

    From Regional Patient Advocate Lim Yong Hao

    Over the past few months, I’ve been reminded repeatedly of the importance of connecting with others in patient advocacy and public awareness work. There’s something truly powerful about meeting and interacting with people in person. Even virtual interactions, whether via video conferencing or email, can still create meaningful connections. You may wonder, “What positive outcomes can come from these interactions?” The answer is simple: plenty.
    One of the most fundamental benefits is knowing that you’re not alone. For patients, it’s comforting to realize that others out there share your experiences. For patient advocates, seeing others who are equally committed to improving patients’ lives is encouraging. Meeting like-minded individuals creates opportunities to support one another, providing motivation on our shared journeys.

    Another significant benefit of connecting with others is the exchange of new information. There are always new developments in the understanding, diagnosis, treatment, and management of HAE. Sharing this knowledge can be incredibly valuable, whether on a personal level or for advancing the work of Member Organizations (MO). One of the most impactful opportunities is collaboration—working together to amplify the impact of our efforts.
    In late June, HAE China held its National Patient Meeting in Beijing. More than 120 patients and family members attended, with many meeting others like themselves for the first time. Patients and families shared their stories, provided mutual support, and maintained a sense of optimism about the future of HAE care in China.

    The meeting included presentations from physicians and patient advocates, including HAEi’s Fiona Wardman. Attendees gained insights into HAE, treatments and upcoming therapies, research, gene therapy principles, and managing gastrointestinal symptoms and laryngeal edema. I was honored to have played a small role in shaping the agenda and identifying additional funding sources, which allowed more patients and families to attend. Unfortunately, due to an accident that left me with a broken ankle, I had to cancel my trip at the last minute. While I was disappointed to miss the event, I was thrilled to hear it was a resounding success.

    During my recovery (while worrying whether the injury might trigger an attack—which fortunately didn’t happen), I was fortunate to participate in two news features. One was a student project for a mass communication program in Singapore. The other was a video recording of my story on a regional medical news platform where I shared insights about HAE and my journey. These opportunities arose through my ongoing connection with the Rare Disorders Society of Singapore. The video was released on YouTube in late October and has received nearly 6,000 views. I’m glad that my story reached both students in the program and a larger regional audience. Surprisingly, the video also sparked interactions in the comments section and private messages, leading to meaningful discussions on HAE. This experience illustrates how connections can lead to opportunities, generating even more connections.

    The 2024 HAEi Global Leadership Workshop (GLW) in Copenhagen was a culmination of this theme of connection. It offered countless opportunities to connect. I reconnected with many patient leaders from my region. I also met patient leaders from Japan and South Korea, whom I am now supporting as their Regional Patient Advocate (RPA) after taking over from Fiona in September. I met patient leaders from Thailand, the Philippines, Pakistan, and India, supported by my colleagues Tony, Henrik, and Fanny (our latest addition to the HAEi RPA Family). Fanny lives in Singapore, so I now have a compatriot nearby for advice and second opinions. I also reconnected with physicians and spoke with representatives from pharmaceutical companies to explore the latest in HAE treatments and potential funding opportunities.

    Aside from the exciting program, the workshop’s highlight was the breakout session I facilitated for East Asia, Southeast Asia, and South Asia patient leaders. I began engaging with MOs three weeks before to gather input for the session agenda. I received valuable insights from everyone, including those from Bangladesh, Afghanistan, Hong Kong, and Indonesia who couldn’t attend. During the session, I shared an overview of the current HAE landscape in the region, and patient leaders from Japan, South Korea, China, Taiwan, Singapore, Thailand, the Philippines, India, and Pakistan shared their plans for 2025. This led to a fruitful discussion on areas where MOs could collaborate. By the end, we collectively identified three critical areas for joint efforts: (1) raising awareness and educating healthcare professionals about HAE, (2) securing funding from pharmaceutical companies, and (3) addressing mental health challenges and stigma faced by HAE patients. I’ve received anecdotal feedback that some leaders took back valuable ideas to implement in their countries, which is a great outcome.

    Our challenge is translating these collaboration areas into actionable plans involving as many MOs as possible. Progress in this area will be testament to how connecting with others can lead to meaningful collaborations. Ensuring patient leaders stay connected will help sustain the momentum and lead to more effective advocacy for HAE.

    Planning for our next opportunity to connect is already underway. The 2025 HAEi Regional Conference APAC will occur from March 7-9, 2025, in Manila, Philippines. Some of the ideas MOs discussed during the breakout session will be integrated into the conference agenda. This event will also include a larger group of patients and caregivers, extending beyond the leaders who attended the GLW. We hope to welcome as many people from the region as possible to the conference; let’s make the most of this opportunity for patients and caregivers to connect and support one another.

    >> Meet the Regional Patient Advocates

  • News from Sub Sahara Africa

    From Regional Patient Advocate Patricia Karani

    The recently concluded 2024 HAEi Global Leadership Workshop was very exciting for those who participated from Sub Sahara Africa. We had patients, doctors, and clinical officers from Mozambique, Comoros, and Kenya attend. The participants were very active, engaged, and eager to learn more about how to provide support for HAE patients in their countries. A highlight from the meeting was the breakout session, where we got a chance to meet and get to know each other on a personal level. The main discussions revolved around raising awareness, early diagnosis, treatment, and better management of the condition. We discussed limited resources and the lack of adequate knowledge in the countries, which is a primary challenge for all countries in this region. We also discussed possible solutions and how we could overcome some of these challenges based on available resources. We factored in questions and answers, which took most of the time during the breakout sessions. The participants reiterated that they had learned a lot from the main conference sessions and were particularly excited about the new HAEi FocalPoint tool, which was easy to join. The countries advised that it would be a very important tool to support their advocacy work; as a “one-stop-shop,” every tool they need in one application. The group was very vibrant and engaged in this session, and we found ourselves running over the time limits for the meeting. Participants appreciated this session, and we formed a communication link to keep in touch.

    >> Meet the Regional Patient Advocates

  • News from South Eastern Europe

    From Regional Patient Advocate Natasa Angjeleska

    It is always exciting to prepare and participate in HAEi’s meetings, but the Global Leadership Workshops that bring so many people connected by HAE in one place is one of the most exhilarating events. There was a large number of interested participants (Member Organization (MO) leads, board members, patients, and caregivers) who applied to come together for the 2024 HAEi Global Leadership Workshop (GLW) in Copenhagen, but also tremendous interest from many physicians to participate in the first ACARE Global Angioedema Forum 2024 (GAF), delivered in partnership with the ACARE network. The packed program for both events was zestful but inspiring. The buzz in the venue may seem confusing for outsiders. But for us, it was an opportunity to immerse ourselves in a way to consume as much information and meet with as many people as possible to make the most of the 2-day meeting.

    During the breakout session, I was eager to get together with participants from SEE: Albania, Bulgaria, Croatia, Cyprus, Greece, Kosovo, Montenegro, North Macedonia, Serbia, Slovenia, Romania, and Türkiye. In June 2024, the MO leads participated in the first-ever State of Management of HAE in the SEE survey. The data was ready to be presented during the breakout. More than 70 representatives in these sessions had an opportunity to converse about the results presented. The group discussed the different advocacy opportunities where they could use this data. The State of Management results offer comparable data between various countries in different areas: diagnosis rates, HAE medications, challenges faced by people with HAE, knowledge of HAE by patients and physicians who treat HAE, and how people with HAE view the government health priorities. In addition, the State of Management presentation supports discussion based on cases showing the burden of not treating HAE: quality of life and cost.

    After the presentation, we discussed topics suggested by the region MO leads before the GLW. The most exuberant discussion was about the opportunities to provide psychosocial support to HAE patients and caregivers. The focus was that the solution was not a MO-organized one-time event or a workshop (although helpful) but continuous support at an institutional level. Greece and Croatia shared good practices. A Bulgarian representative raised an interesting suggestion for organizing forum discussions on a regional level on different topics, such as parents of male or female patients with HAE – how to speak with kids at different ages on issues relevant to particular developmental stages or how to support your spouse, or how to deal with challenging discussions in relationships, etc. Many ideas were bubbling in the room, and we agreed to organize future online meetings to plan the delivery of some of these exciting ideas. After the GLW, I received many messages supporting the concept of an inter-regional meeting and expressions of gratitude for the opportunity to contribute to the meeting via the breakouts.

    This GLW also introduced Masterclasses on different topics, using interviews between RPAs and representatives from different countries. I facilitated the Masterclass for Raising awareness through media and events with representatives from HAE Bulgaria Danail Dimov, and HAE Canada Michelle Cooper. At the Masterclass for Fundraising opportunities, HAE Macedonia president Natasha Jovanovska Popovska presented a case study from HAE Macedonia. HAE Türkiye representative Ersan Sevinç participated in a Masterclass about Effective advocacy strategies for local national impact. All these sessions offered so many different experiences and viewpoints, and GLW participants were highly engaged.

    I truly hope each participant was inspired and energized to stay active and navigate the future for an improved quality of life for all people with HAE and their family members.

    >> Meet the Regional Patient Advocates

  • News from Central Eastern Europe and Middle East

    From Regional Patient Advocate Michal Rutkowski

    It likely won’t come as a surprise that my recent activities have been around the biggest HAE event of the year – the 2024 HAEi Global Leadership Workshop (GLW). I had the great privilege of actively participating in this gathering, and I’d like to share the key highlights relevant to the regions I am responsible for, and some additional updates from Copenhagen and beyond.

    The GLW is a flagship event for the global HAEi community, offering a unique opportunity to engage with leaders from patient organizations, expert physicians, and representatives from industry partners. I would like to extend my deepest gratitude to all the HAEi Member Organizations for their unwavering dedication and efforts to attend. I am incredibly proud that the following countries from Central Eastern Europe and the Middle East were able to be present in person: Armenia, the Czech Republic, Egypt, Georgia, Hungary, Iran, Kazakhstan, Lithuania, Oman, Poland, Qatar, Saudi Arabia, Slovakia, Ukraine, and the United Arab Emirates. I truly hope that those unable to attend this GLW will be able to join us in the future.

    The GLW is a testament to how individuals from different cultures, ethnicities, and languages – each bringing their unique perspectives – can come together in a shared mission to improve the global outlook for patients with HAE. We gathered in Copenhagen twelve years after the first HAEi Global Conference and witnessed the remarkable progress made in just over a decade.

    RPA Maria Ferron and I decided to group culturally similar regions for more effective breakout discussions. This approach allowed us to host two separate breakout sessions: one for the Middle East (ME) and North Africa (NA) and another for the Mediterranean, British Isles, Benelux, and Central Eastern Europe. Below, I’ll share what we accomplished during the session for the ME and NA regions.

    Maria and I decided to initiate the HAEi Patient-Driven Research program called the “State of Management of HAE” to create a report on the current state of care and medication access for HAE patients in ME and NA. Working with Debs Corcoran, HAEi’s Chief Scientific Officer, and Mohamed Osman, HAEi Advocacy Facilitator MENA, we designed and translated the survey. Member Organizations (MO) completed it during the session. HAEi will analyze the responses to produce the final report, which will be the first of its kind from HAEi for ME and NA. This study is a crucial first step in assessing the situation in individual countries and regions, and it will serve as a foundation for future engagement with health ministries across the ME and NA regions.

    During the session, we provided participants with valuable insights into clinical trials, specifically focusing on HAE trials. This topic is particularly relevant given the growing interest in conducting these trials in Middle Eastern countries. We also addressed the potential for cross-border clinical trials, highlighting how the linguistic and cultural similarities across the ME and NA regions could facilitate patient participation in trials conducted outside their home countries, depending on local regulations. This could enhance the region’s involvement in clinical research, accelerate the registration of new therapies, and improve patient access to innovative treatments.

    Additionally, we presented participants with an overview of the modern HAE medicines currently registered/licensed in ME and NA. We also encouraged MOs to run active social media awareness campaigns, utilizing multilingual materials–available in Arabic and French–prepared by HAEi. This approach enables MOs to efficiently and effectively reach a broader audience, increasing awareness and engagement with HAE patients in their respective countries.

    Finally, we explored several HAEi tools to support MOs and individual patients. These included HAEi FocalPoint, HAEi Connect (now available in Arabic), the Patient Travel and Relocation Guide, and the Virtual Meeting Toolkit.

    The session was marked by engaging questions, lively discussions, and a shared sense of accomplishment, with participants feeling that the time spent was valuable and productive.

    In addition to my involvement with the GLW, I was actively engaged in various initiatives supporting the MOs from the regions under my responsibility. Below are some of the key activities:

    • I was privileged to conduct virtual meetings with representatives from Armenia, Georgia, Hungary, Iran, Jordan, Kazakhstan, Lebanon, Russia, Ukraine, and the UAE. During these discussions, we addressed the current challenges faced by our members, and together, we worked towards finding solutions to some of these issues.
    • I collaborated with HAEi’s Chief Scientific Officer, Debs Corcoran, as well as fellow RPAs Natasa Angjeleska, Maria Ferron, and Jorn Schultz-Boysen on the HAEi Patient-Driven Research: State of Management of HAE in Europe.
    • With the launch of the next edition of the LEAP educational program, I reached out to MOs, encouraging them to collaborate with young people from their respective countries.
    • Supporting local HAE communities remains a top priority for HAEi. In recent months, alongside HAE Jordan and Mohamed Osman, we have invested significant effort in organizing another local community meeting, this time in the capital of Jordan, Amman.
    • We continue to work to broaden access to HAEi’s tools and resources:
      • “Women with HAE” has recently been translated into Arabic, Czech, Kazakh, Lithuanian, and Russian. We are also progressing with the Persian translations of the “Understanding HAE” and “Women with HAE” booklets.
      • We are collaborating with HAE Slovakia to develop and host their new website.

    >> Meet the Regional Patient Advocates

  • News from Mediterranean, North Africa, Benelux and British Isles

    From Regional Patient Advocate Maria Ferron

    The months leading up to the 2024 HAEi Global Leadership Workshop (GLW) have been nothing short of exhilarating. Coordinating the participation of representatives from various countries is one of the more challenging aspects of preparing for the GLW. However, seeing how effectively we can bring together diverse voices and perspectives is incredibly rewarding. I’m delighted to report that we have secured a good representation from most countries in the region. In the weeks leading up to the GLW, I immersed myself in developing content aimed at addressing the pressing issues HAE patients face. The excitement of preparing presentations and discussions that promise to foster dialogue and inspire positive change has been invigorating.

    During the GLW, HAEi dedicated time to a series of regional breakout sessions. Fellow RPA Michal Rutkowski and I decided to group culturally similar areas from our regions for more effective breakout discussions. This approach allowed us to host two separate breakout sessions: one for the Mediterranean, British Isles, Benelux, and Central Eastern Europe countries, and another for Middle East (ME) and North Africa (NA).

    Among the many highlights of the GLW, the breakout sessions stood out as pivotal settings for in-depth discussions and actionable strategies. The Mediterranean, British Isles, Benelux, and Central and Eastern Europe session was structured around key themes relevant to these areas. The informal atmosphere encouraged networking, allowing leaders in the HAE community to forge valuable connections. Many left the breakout with plans for the upcoming months, for example, implementing some of the tools and resources HAEi offers to all the Member Organizations, being prepared to manage their HAE, or being more active on social media to advocate and raise awareness. The energy and passion exhibited by the participants during the breakout sessions emphasized the collective commitment to making meaningful progress in the field of HAE.

    Outside of the GLW preparations, the summer season brings unique challenges for patients with HAE, making it crucial for me to ensure that patients enjoy their vacations and do so with the necessary medical preparedness. Amid bustling tourist activities, I prioritized connecting with patients to ensure they have comprehensive emergency plans in place. Establishing communication with local healthcare providers and regional associations is essential to facilitate timely assistance if needed. Additionally, I encourage patients to utilize the resources available through HAEi. The “HAEi Travel & Relocation Guide” is an invaluable tool that offers essential tips for safe travel and relocation, catering specifically to those living with HAE.

    >> If you’re seeking further information, check it out here.

    This period has also provided me with the opportunity to support countries grappling with access to modern treatments for HAE. Advocating for these advancements is crucial to improving the quality of life for patients in our region. Beyond that, I am thrilled to share that we’ve made significant strides in the official registration of the Belgium association. This milestone marks progress for Belgium and enhances our collective efforts to promote awareness and understanding of HAE worldwide.

    Returning to the GLW, the workshop provided a dynamic platform for HAE leaders to collaborate, share insights, and drive initiatives to improve patient care and advocacy. I can hardly wait to see what the future holds as we progress together after this important event.

    >> Meet the Regional Patient Advocates

  • News from Nordics

    From Regional Patient Advocate Jørn Schultz-Boysen

    The highlight of recent months has undoubtedly been the 2024 HAEi Global Leadership Workshop (GLW) and the inaugural ACARE Global Angioedema Forum 2024 (GAF), a pivotal moment for the global HAE community.

    I had the privilege of co-hosting a breakout session alongside Fanny Schappler. We convened the leadership of the HAE organizations from Germany, Austria, Switzerland, Finland, Sweden, Norway, and Denmark.

    The 3-hour breakout session provided a valuable opportunity for everyone to share their experiences, particularly regarding access to medication, which can be a significant challenge even in generally well-resourced countries. One story, for example, highlighted how where a patient lives within a country can dramatically impact their treatment options. These discussions were a strong reminder of why our work is so important, no matter how developed or resourceful a country may seem.

    We also explored new HAEi tools, including the HAEi Patient Travel & Relocation Toolkit and the HAEi Virtual Meeting Toolkit. In addition, we touched on many of HAEi’s established resources—such as Hosted Websites, Connect, FocalPoint, Women with HAE, Understanding HAE, LEAP25, and Advocacy Academy—all of which sparked significant interest. It was encouraging to see how eager the participants were to explore and utilize these tools to benefit their communities.

    Thank you to everyone who contributed to this discussion and for your vital work in your respective country. I left the workshop feeling inspired. While the challenges faced by people with HAE and their families are ongoing, the commitment and passion within our community give me immense confidence in the path ahead.

    I have had the privilege of working with the region covering Germany, Austria, Switzerland, Israel, Finland, Sweden, Norway, Denmark, and Iceland for several years. However, as of 1 September 2024, we restructured these duties. My colleague Fanny Schappler has taken over responsibilities for Germany, Austria, Switzerland, and Israel. Fanny was introduced to these Member Organizations before Copenhagen and had the opportunity to meet many of them during the workshop. I know she is excited to continue the great work in this region.

    Thank you to all the wonderful people I have worked with in Germany, Austria, Switzerland, and Israel. I look forward to meeting you all again soon, and I am confident you will achieve wonderful results with Fanny’s support.

    I am eager to continue this critical work as I now focus on my new region—Finland, Sweden, Norway, Denmark, Iceland, Greenland, and the Faroe Islands. I look forward to the progress we will make together. Thank you again to all who participated in this impactful GLW, and I look forward to continuing our efforts on behalf of the HAE community.

    >> Meet the Regional Patient Advocates

    We continue recruiting patients and caregivers for the ongoing Pharmaco- and Socioeconomic study in Germany, Austria, and Switzerland.

    This important survey focuses on how access to treatment impacts on quality of life—not just for the individual with HAE but their entire family.

    We encourage as many patients and caregivers as possible to participate to ensure a comprehensive and impactful study >> click to participate

  • News from Oceania and South Africa

    From Regional Patient Advocate Fiona Wardman

    To enhance support across the Asia-Pacific (APAC) region, I have transitioned some of my responsibilities to my colleagues, Yong Hao and Fanny, the RPA leads for other APAC region countries. I focus on supporting Australia, New Zealand, South Africa, and other Oceania countries, ensuring they continue receiving the guidance needed for growth and development. This redistribution allows us to tailor our efforts more effectively to meet the unique needs of each country.

    Uniting Leaders at the 2024 HAEi Global Leadership Workshop (GLW) Breakout Session
    During my GLW breakout session, we brought together leaders from my group of countries—Australia, New Zealand, South Africa, as well as those from Canada and the USA. This gathering was a valuable opportunity for these countries to exchange knowledge, discuss key challenges, and develop collaborative strategies to strengthen their organizations. Leaders from each country actively shared their ideas, challenges, and diverse experiences, contributing to an inclusive and highly interactive environment.

    A highlight of the session was the introduction of two new HAEi resources: the HAEi Patient Travel & Relocation Guide and the HAEi Virtual Meeting Resource. HAEi developed these tools to support patients navigating relocation challenges and to help organizations improve their virtual engagement practices. The reception to these resources was overwhelmingly positive, with leaders recognizing their potential to benefit their respective communities significantly.

    A key focus during the discussions was on expanding outreach using modern approaches. Leaders emphasized utilizing social media platforms to amplify awareness, share impactful patient stories, and spread valuable resources. Ideas like offering internships and creating engaging podcasts were also explored as fresh ways to connect with broader audiences and draw attention to advocacy efforts.

    Participants highlighted successful advocacy campaigns that led to discovering new patients and increasing collaboration with medical professionals. The group also tackled common hurdles such as resource limitations and youth engagement barriers, sharing practical solutions to address these challenges.

    Despite the differences in size and capacity among the participating organizations, the collaborative spirit during the breakout session was truly remarkable. The collective brainstorming led to new insights and practical strategies that everyone felt confident could be applied within their countries. Leaders left feeling energized and equipped with new strategies, ready to drive greater impact in their respective countries.

    Looking forward
    This GLW breakout session underscored the power of global collaboration in tackling shared challenges. Moving forward, leaders from Australia, New Zealand, South Africa, Canada, and the USA remain deeply committed to advancing volunteer engagement, strengthening advocacy efforts, and increasing organizational capacity.

    The transition of countries to different RPAs and the success of all the GLW breakout sessions have set a strong precedent for ongoing growth in each HAEi region. By harnessing the collective expertise and shared dedication of all MO leaders, we are on a solid path toward making a lasting impact in the communities we serve.

    >> Meet the Regional Patient Advocates

  • News from Central America and Caribbean

    From Regional Patient Advocate Javier Santana

    During the 2024 HAEi Global Leadership Workshop (GLW), patients from my region – Central America and Caribbean – were able to share ideas and strategies with patients and leaders from other countries and listen to the different talks and experiences shared on the main stage by healthcare professionals, other patients, and HAEi representatives.

    In the breakout session, group leaders from the countries I support talked about the current HAE situation in their countries, what the issues are, and how they are working on overcoming them. Additionally, leaders exchanged ideas and strategies on how they can support each other and work together to achieve their future goals.

    I also used the breakout to explain in more detail how the different programs and tools HAEi offers work, including HAEi Advocacy Academy, HAEi Connect, HAE TrackR, HAEi FocalPoint, and the new HAEi Travel and Relocation Guide.

    During the main program, Tayra Corrales from Costa Rica and I gave a talk to the audience about Strategies for Engaging Government and Health Ministries. The authorities in Costa Rica recently approved two medications for HAE thanks to efforts made by patients with HAE, non-profit organizations, and HAEi. During the talk, Tayra Corrales spoke about their experiences during the authorization and reimbursement process. I also gave a talk on advocating for the creation or promotion of laws on HAE to guarantee the right to access treatments, create more awareness about HAE, and protect patients’ rights. You can read more about these talks in ‘Getting political to get results’ and see the “The power of the law – to help your advocacy efforts” feature in Global Perspectives July 2024.

    Patients were grateful for the workshops offered and the access to pharmaceutical representatives who could speak with them directly to answer their questions or concerns about different HAE medications currently available globally.

    Physicians from my region who attended the ACARE Global Angioedema Forum 2024 (GAF) also expressed their appreciation for the information and research shared by the expert HAE speakers. Many of them have shown great interest in certifying and promoting the creation of ACARE Centers in their respective countries.

    I hope all attendees enjoyed the GLW and GAF, and I look forward to seeing you at the next conference.

    >> Meet the Regional Patient Advocates

  • News from South Asia, Germany, Austria, Switzerland, and Israel

    From Regional Patient Advocate Fanny Schappler

    My journey as a Regional Patient Advocate (RPA)

    I officially began my journey as a Regional Patient Advocate (RPA) in September 2024, supporting the following countries: Germany, Switzerland, Austria, Israel, India, Sri Lanka, Bangladesh, Nepal, Pakistan and Afghanistan. Stepping into this role felt exciting and daunting, but I was eager to continue the meaningful work already established and carve my own path forward. I am grateful for the invaluable support of my fellow RPAs and the Leadership Team, who provided guidance and encouragement as I settled into the role.

    In the past two months, my focus was the following areas:

    • To learn from each MO and its leaders
    • To understand their unique challenges and opportunities
    • To get to know their personal story with HAE
    • To understand how far each MO is on its advocacy journey.

    It’s important for me to understand what value I can bring to each of my MOs, and I want to be an enabler. I support a variety of countries with differing healthcare landscapes; the journey of a patient from first symptoms to an established diagnosis and having a proper treatment plan in place is vastly different in each country. I feel it is critical for me to acknowledge this and first hear what objectives each MO has for the future so I can best support them along the way with the numerous tools HAEi has.

    Having a helicopter view is one of the values the RPA can bring. I can identify best practices from country X and suggest them to country Y. Sometimes, there is no need to reinvent the wheel, and we can learn so much from the journeys others have paved before us!

    In parallel to getting to know each country and its unique healthcare landscape, I also completed all the training modules available at HAEi Advocacy Academy during my first month. It’s incredibly resourceful, and I love the one-stop-shop approach and small bites on which each module is structured. It makes the information easy to digest and remember. I strongly recommend these modules to any of our members who want to step into advocacy!

    What was the 2024 HAEi Global Leadership Workshop (GLW) like for you?

    The GLW was nothing short of an exceptional experience. It provided a unique opportunity to connect with other RPAs, MO leaders worldwide (90+ countries represented!), and doctors from the countries I support. The interactive sessions and discussions were inspiring, and my favorite part was the case studies from MOs. Often enough, the challenges encountered in building and growing a patient advocacy group are similar. It can be daunting, and I imagine one may feel alone sometimes. Yet when you realize others have been in the same spot, embraced the challenges, and turned them into opportunities, it gives you the strength and motivation to reach your goal despite the obstacles along the way. As in many life situations, advocacy work is not a sprint but a marathon!!

    What do you hope to achieve as an RPA for your region?

    As an RPA, my primary goal is to build the capacity of the existing MOs in South Asia (some relatively new) and support a sustainable future for the long-standing MOs in DACH countries. In the short term, I hope to help the MOs increase their members and ensure they build the relevant infrastructure to be prepared for achieving their mission and growing along the way. In the long term, I am committed to ensuring all healthcare players know what HAE is and the importance of a diagnosis and management plan, as well as ensuring that our role as patient advocates is recognized for its central role in shaping policies. Which ultimately will lead to improved patient outcomes and a better life for persons living with HAE and their loved ones!

    >> Meet the Regional Patient Advocates

  • News from South America and Mexico

    From Regional Patient Advocate Fernanda De Oliveira Martins

    The past months were of preparation for the 2024 HAEi Global Leadership Workshop (GLW); preparing some Member Organization (MO) leaders for participation in the GLW program, translating the last guide for the participants, and planning the activities for the South America and Mexico breakout session. While working towards the GLW, I could still assist the MOs in implementing HAEi tools, including developing the Emergency Department poster tailored for Venezuela, and supporting the advocacy for modern HAE treatments by participating in public consultations in Brazil.

    At the GLW, I led the South America and Mexico breakout session. The goals: foster connections, learning, and fun! We were a group of around 70, some old friends who were seeing each other again, and meeting new people. For the activities, we split into 10 teams with people from different nationalities in each team. The afternoon began with MO leaders reflecting on successes, challenges, and future ambitions. A balloon activity highlighted problems and opportunities, fostering collaboration and initial steps toward goals. We concluded with an enjoyable quiz to test knowledge and to learn more about the disease, HAEi, and its tools. We thoroughly enjoyed the group’s energy, each leaving recharged to achieve more in their own countries.

    >> Meet the Regional Patient Advocates

Navigating the Future: 2024 HAEi Global Leadership Workshop (GLW) and ACARE Global Angioedema Forum 2024 (GAF)

From 3 to 6 October 2024, around 750 HAEi Member Organization board members/leaders, physicians/scientists, and industry representatives from 90 countries met in Copenhagen.

GLW
The GLW was tailored to reflect feedback from previous meetings. You wanted more networking, more breakouts, more experiences, more capacity-building information (to name but a few). We considered all your feedback from previous meetings and here you can read how this was delivered.

“We listened, we heard you, and we want to provide the best possible support and education to make you the best possible advocate.”

– Fiona Wardman, HAEi’s Executive Vice President Global Advocacy and Chief Diversity Officer 

GAF
The first GAF meeting set the bar for future meetings. You can read our summary of the meeting and find the abstract book here.

GAF Meets GLW
A unique opportunity for healthcare professionals, patient leads from advocacy organizations, and industry representatives join together to inspire each other. Read all about it here.

  • Welcomes and goodbyes

    Every seat was taken, but someone was missing. A record-breaking 750 healthcare professionals, patient leaders, and industry representatives packed into a vast room in Copenhagen, yet one absence was noted.

    Professor Marcus Maurer, inspirational clinical leader, ally, and friend to the global HAE community, died in a tragic accident in late July 2024. As HAEi’s Chief Medical Advisor and the driving force behind the scientific forum of this meeting, Marcus would have opened proceedings. Instead, his colleague and friend Professor Markus Magerl stood on stage with HAEi’s Tony Castaldo and Henrik Balle Boysen.

    Professor Magerl opened his tribute to his friend by acknowledging it should have been Marcus on stage. Prof. Magerl shared how Marcus ended up studying the mast cell and how he’d remained loyal over all the years, describing Marcus as an outstanding clinical allergy researcher and one of the most brilliant minds in dermatology and allergology. Prof. Magerl told the audience that Marcus’ contributions were so significant that angioedema research would be unthinkable without him, ‘He was in a class of his own.’

    Speaking to Marcus’ dynamism and drive, Prof. Magerl felt that for Marcus, there must have been more than 24 hours in his day because there were no areas in which he wasn’t active. Marcus led the development of national and international guidelines for angioedema; he developed diagnostic devices; he found new ways to assess disease activity and quality of life; and above all, thanks to his immense perseverance, we now have effective drugs and more to come.

    Beyond his clinical achievements, Prof. Magerl painted a picture of a unique and extraordinary person. Talking about his ‘dear colleague and loyal friend,’ Prof. Magerl, at times his voice breaking with emotion, spoke about Marcus as a man who was always communicative, friendly, and never condescending. He allowed others to shine and was happy about it, Professor Magerl told the hushed audience. He concluded by asking all present to continue Marcus’ legacy.

    At the end of his emotional eulogy, the audience offered their tribute with a prolonged standing ovation.

    You can read more about Marcus Maurer’s life in our Memories of Marcus feature.

    After thanking Prof. Magerl for his inspiring tribute, Tony Castaldo told the audience that the best way to honor Marcus Maurer’s remarkable academic and advocacy efforts was to continue the programs and activities he initiated.

    Welcoming all parties to the joint 2024 HAEi Global Leadership Workshop (GLW) and ACARE Global Angioedema Forum 2024 (GAF), Tony and Henrik drew attention to the generous support of the pharmaceutical company sponsors, and how this demonstrates the shared commitment to collaboration, which is such an important element of these meetings.

    Both groups would unite in a unique cooperation between the physician and patient communities when “GAF meets GLW” to Navigate the Future.

    Tony and Henrik ended their welcome by dedicating the event to the enduring legacy of Prof. Marcus Maurer, whose leadership and passion will continue to inspire everyone and to follow Marcus’ mantra, ‘Never Give Up.’

  • Memories of Marcus

    The death of Professor Marcus Maurer is a profound loss to everyone in the HAE community, whether they are a scientist, patient, or caregiver. His dedication to improving the lives of people with angioedema was total. However, despite touching countless lives through his work, there will be many people who never met or interacted with him. Here, we try to show a little more of Marcus’ character by sharing personal reflections and anecdotes from those who knew him best.

    For our own part, the editorial team interacted with Marcus on numerous occasions. He was always a great interviewee, offering lively quotes. However, it is his almost limitless energy that we remember most. In 30 years of attending scientific conferences, we had never seen a clinical expert leap on and off the stage during presentations, until seeing Prof. Maurer in action.

    Henrik Balle Boysen
    Marcus was an incredible person and a truly warm-hearted friend you could always count on. His interpersonal skills were remarkable, and he made sure to include everyone in everything he did. But beyond his kindness, Marcus was defined by his unwavering determination to “Never Give Up!” I remember our interactions as he traveled to our recent regional conference in Panama. With strikes across Europe canceling flights left and right, most would have called it quits. Not Marcus. He embarked on a long car ride, caught a train, and navigated through several connecting flights. Though delayed by nearly 20 hours, he arrived just in time to participate, a testament to his perseverance. The loss of Marcus is beyond words, and he will be deeply missed. Rest in peace, my friend.

    Tony Castaldo
    The HAEi community has always known that our Chief Medical Advisor Prof. Marcus Maurer possessed a set of extraordinary talents. A recent experience prompted me to add ‘magician’ to his amazing legacy. Not long before I was due to go on stage at the 2024 HAEi Regional Conference Americas, the hotel front desk called to say the laundry contractor could not locate the jacket I had sent to be cleaned. As I got a microphone fitted right before going on stage, Marcus apparently overheard me telling the sound technician how I like wearing a jacket when on stage and couldn’t this time because the hotel had somehow lost it.

    Marcus came over to me and said, ‘Don’t worry, gotcha covered.’ I offered a smile acknowledging the kind thought, but was already in my own world anticipating how I would deal with being on stage feeling incompletely dressed.

    A few moments later, I started my talk and made a quick joke about my missing jacket. Then, I heard a familiar, but totally unexpected voice stop the show. It was Marcus saying, ‘Hold on, Tony, I have something you were looking for!’ He proceeded to jump on the stage with my jacket and with great fanfare helped me put it on.

    Right after my talk, I stopped by the front desk to thank the people I had spoken to earlier for locating my jacket. They just stared at me while I spoke and finally said, ‘Sir, our vendor is still looking for your jacket, which is still showing as lost in our system.’ I walked away chuckling and thinking… is there anything the amazing Marcus Maurer can’t accomplish? And here is the kicker! I was so mesmerized by the “magic” of the moment, I had no desire to ask how or where he found that jacket.

    Prof. Marc Reidl
    Marcus always greeted people with a warm smile. He had a remarkable ability to make everyone feel they were now ‘among friends’ even if he was meeting you for the first time. I remember a familiar look Marcus would give when he knew something you didn’t know. I saw that look a lot and it meant he was about to share something important, interesting, or clever. Marcus was the consummate teacher and collaborator, but above all, a wonderful friend. He will be deeply missed.

    Prof. Markus Magerl
    Marcus, Thomas, and I recently wanted to run an angioedema school in India. Marcus and Thomas traveled together from Berlin and we were to meet up in India. Unfortunately, they got stuck in Istanbul and couldn’t get any further.

    The angioedema school took place anyway because they spontaneously recorded all their presentations and contributions on video at the airport in Istanbul and sent them to the organizers. Their contributions, totaling several hours, were available just in time for the event’s start. Marcus was a master at solving small and large problems quickly and in the best possible way. Canceling the event would have been out of the question for him. Never give up!

    Prof. Konrad Bork
    We mourn the loss of Prof. Marcus Maurer, who died before his time. He was taken from us by a tragic and unbelievable fate. We miss Marcus. We miss our friend with his drive and his cheerful, beaming personality. For many years he was full of ideas, plans, and projects. No one can fill his place.

    Everyone felt his vital magnetism when he spoke privately or publicly about his new projects. His natural kindness and his ever-present energy enabled him to involve a large number of people and scientists in his projects. We will miss his inspiration and enthusiasm. Marcus brought our community closer together, more than ever before, for the sake of the patients with recurrent angioedema.

    Condolences and further memories
    All at Global Perspectives, and the wider HAEi team, would like to extend their sympathy to Professor Maurer’s family and friends. A book of condolences can be viewed and signed at marcus-maurer.com

  • An accessible update

    ‘We have achieved something unprecedented for an ultra-rare disease,’ Henrik told the assembled patient advocacy leaders at the beginning of the 2024 HAEi Global Leadership Workshop, with eight treatments approved and new options being studied. However, Henrik recognized that this access was not spread evenly, with patients in many countries only having access to anabolic steroids and tranexamic acid. This workshop, he said, is designed to win the fight for access by equipping every participant with the tools and skills needed.

    A vital part of winning the fight for access is equipping every participant with the tools and skills needed. Henrik stated the first step was knowing what was out there and turned to Tony Castaldo for a review of currently available and forthcoming therapies.

    Tony began by reminding all present they are an advocate and their goal is to improve the quality of life for people with HAE. The route to this is winning access to and reimbursement for modern HAE medicines. A crucial element of advocacy, Tony counseled, was to ‘be aggressive.’ He called on all patient advocates to interact with pharma company representatives to make sure their voices were heard and to not be afraid to ask for commitment and support.

    Tony outlined the medicines available, noting that information can vary by country and that you should consult the prescribing information for your country for specifics.

    >> Find a list of licensed medicines on haei.org

    Looking to the future, Tony described the ‘astonishing level of pharmaceutical company interest in the next generation of HAE treatments,’ which resulted from ‘our motivated and united HAEi community’ and the consistent and proactive role in Navigating the Future.

    Tony then presented information on the six companies and medicines being tested, which called for some very challenging pronunciations.

    >> Find the list of HAE treatments in development on haei.org

    Tony closed the session by praising the unselfish work of all who pave the way for a brighter future by participating in clinical research and the can-do attitude of our community.

  • Getting to know your Regional Patient Advocate

    Fiona Wardman, HAEi’s Executive Vice President Global Advocacy and Chief Diversity Officer, took to the stage to introduce the incredible Regional Patient Advocates (RPAs) that support Member Organizations (MO).

    Through a light-hearted Q&A, the audience discovered that (left to right):

    Henrik Balle Boysen, with responsibility for South East Asia: Became a ski guide without ever skied; when you’re desperate enough for a job, you’ll find a way.

    Jørn Schultz-Boysen, on home turf today, representing the Nordics: Admitted to being a San Franciso 49ers fan.

    Michal Rutkowski, looking after Central Eastern Europe and the Middle East: Has indeed grown from small beginnings as a very premature baby, weighing just 1.88kg.

    Patricia Karani, taking care of Sub Sahara Africa: Revealed her love of cooking and its ability to help her to relax.

    Natasa Angjeleska, working with South Eastern Europe countries: Showed her competitive instinct by coming third in a recent paddleboard event.

    Yong Hao Lim, supporting the East Asia region: Showed his dancing feet with an impromptu Argentine tango.

    Fanny Schappler, covering Germany, Switzerland, Austria, Israel and South Asia: Drinks Ovalmaltine, a unique malted drink beloved of Swiss people, every day for breakfast; ‘It’s an addiction,’ she said.

    Maria Ferron, Mediterranean, North Africa, Benelux, and British Isles: Would love to meet the naturalist David Attenborough.

    Fernanda De Oliveira Martins, looking after South America and Mexico: Was an elementary school teacher, she taught Brazilian children about science.

    Javier Santana, RPA for Central America and Carribean: Had a childhood of guitar playing and singing but refused to demonstrate his singing prowess on stage.

    Tony Castaldo, representing North America and South East Asia: Recalled a past life as a trainee Catholic priest, performing the Latin mass, including solos.

    Last but not least, Fiona introduced her role as RPA for Oceania and South Africa: And demonstrated her astonishing ability to imitate the national bird of Australia, the kookaburra.

    Fiona concluded the session by describing the RPAs as ‘unbelievable advocates’ working for you, the MOs, every day.

  • Masterclasses in empowering advocacy leadership

    Led by RPAs, the Masterclasses are a series of short sessions that tackle the big issues facing advocacy leaders. In this Empowering Advocacy Leadership masterclass, RPAs covered handling personal data effectively and safely, and becoming a better advocate.

    First to speak was Jørn Schultz-Boysen. With his commercial background in data and IT, Jørn was well-placed to give attendees some simple tips and advice on managing data securely.

    ‘Protecting data is everyone’s responsibility. For MOs, it’s about safeguarding the trust between you and your members.’

    Jørn reminded everyone that data breaches can have devastating effects, financial costs and lead to potential loss of trust from the community.

    Trust only sources you know
    Email and internet fraud are things most people will encounter in everyday life; they are also the most common sources of data breaches. Trust was the most important thing, he reminded the audience. ‘Phishing’ attempts were, Jørn said, not the fun kind where you catch a fish. Red flags include unexpected requests for sensitive data or a sense of urgency. Offers that appear too good to be true usually are.

    Jørn’s simple advice was, ‘Be very careful about who you give your personal data to. Never hand out login details. Buy only from a provider you know and trust. Trust only sources that you know.’

    Jørn was very clear on the importance of passwords. His top tips are:

    • Use strong passwords; Unique combinations of letters, numbers, and symbols constitute a strong password
    • Whenever possible, enable 2-factor authentication (where you are sent a unique code to input)
    • Avoid recycling passwords. ‘This is the only time that recycling is bad,’ Jørn joked.

    Regular software updates protect your data from vulnerabilities that hackers may exploit.

    Jørn’s other top tips for data security were:

    • Never give out your login details
    • Always lock your devices when you’re not using them
    • If something seems suspicious in an email (link or attachment) or on a website, don’t click
    • Use a VPN whenever you use a public Wi-Fi connection
    • Consider an incident response plan (this details what to do if something bad happens, so you recover quickly and effectively)
    • Don’t ask for more data than you need.

    ‘The more data you have, the more you can lose.’
    Jørn Schultz Boysen, RPA

    It’s everyone’s responsibility to be proactive about IT Security. Jørn encouraged all MOs to consider using the free HAEi tools, which have the highest standards of data security built in.

    HAEi FocalPoint is a one-stop tool for MOs. It provides a single point to login to any HAEi tools, and a single place to capture all the core information related to running an advocacy organization, such as treating hospitals, physicians, and available treatments. Inputting into HAEi FocalPoint means that any changes are automatically updated across an HAEi-supported website and communicated directly to your RPA. The tool also acts as a way to handle travel grant applications.

    HAEi Connect, was introduced by Fiona Wardman. She asked for a show of hands from the audience: ‘Who was currently using a Word file, Excel spreadsheet or even paper records with members’ details?’ Thanking everyone for their honesty, she made clear that when people have trusted you with their personal data, those forms of record keeping just weren’t secure enough. HAEi Connect is a free and secure database tool exclusively for HAEi MOs. It is GDPR compliant, which is the highest level of protection of privacy and security.

    ‘I want to know that my information is secure, that my privacy is protected so that no hackers can access my personal data.’

    Fiona gave the audience a whistle-stop tour of HAEi Connect. She started with the dashboard, which shows a quick overview of member data, and any outstanding data updates needed, whether from patients or physicians, media or pharmaceutical contacts.

    Data is at its most valuable when it can be used effectively. Fiona outlined examples such as collecting a cohort of patients for a forthcoming clinical trial or contacting only specific patients affected by a medication shortage. HAEi Connect means you can search and select the right people to contact every time, whether through emails or text messages; whatever works best in your country.

    Fiona highlighted testimonials from HAE Brazil and HAE Peru; both of them felt that it helped them to keep patient data secure and offered a user-friendly tool for communicating with members and with HAEi.

    An Advocacy Academy
    Moving away from data, Michal Rutkowski returned to the stage to discuss the importance of continuing education for advocates, and to showcase the value of HAEi Advocacy Academy. He described it as a powerful, free-to-use online learning platform, designed to support people with HAE in everyday life, and help those in MOs to get the most from their advocacy efforts. Whether you are just starting out or an experienced advocate, there is something for you at HAEi Advocacy Academy.

    HAEi Advocacy Academy offers learning in small, manageable chunks, which can be accessed anytime, anywhere in the world. ‘All you need is internet access,’ Michal quipped.

    ‘HAEi Advocacy Academy lets you learn new skills in your own time and at your own pace.’

    The available courses are split between Open Access and MO Exclusive Access. The first type of course is available to all; you just need to create a free account to get started. For MO Exclusive Access, contact your RPA, who will share your specific code to get you started.

    The Open Access courses focus on HAEi resources, good-to-know tips, a workshop, and a youngsters’ toolkit. The MO Exclusive Access courses go deeper, with learning and skills about advocacy, running an MO, and using HAEi resources.

    Michal concluded by sharing that HAEi Advocacy Academy had helped him become a better leader in HAE Poland and offer better help and support to patients in his home country.

  • BioCryst’s community commitment

    For the first time, a pharmaceutical company took the stage at an HAEi global event; BioCryst, a Diamond Supporter of the GLW, had a big statement to make to the assembled HAE advocates.

    Charlie Gayer, Chief Commercial Officer at BioCryst, and his colleague Kristin Buske, Head of Global Medical Affairs, were welcomed on stage. They started by expressing their gratitude for the partnership with all in the HAE community; all that BioCryst has been able to accomplish was as a result of countless hours of feedback they’ve received from patients and advocates.

    Charlie told the audience that in 2019, half of their company crossed America to spend time with their friends in the HAE community. This event had a profound impact and continues to be cited by BioCryst’s President and CEO, Jon Stonehouse, as a very meaningful moment.

    Since then, BioCryst has been with the HAE community at HAEA National Summits and the HAEi Regional Conferences Americas, APAC, and EMEA. During these meetings, the BioCryst team is often humbled by the interactions and conversations they have with people living with HAE.

    Charlie described hearing many times: ‘Nothing about us, without us.’ The theme that emerged from these discussions: You’re The Key. Everyone in the company is motivated to look for a bright future for HAE, Charlie told the audience, knowing that ‘all of you are the key’:

    “You inspire us every day, and you motivate us to work harder every day for that bright future.”

    Acknowledging that there are now 100 Member Organizations and that BioCryst has reached 500 employees in 13 different locations, it is clear that both the community and the company are growing. The first oral, once-daily, preventative therapy for HAE is now available in over 20 countries, and in the next few years, BioCryst hopes to reach many more.

    At this point, Charlie re-stated a commitment first made by BioCryst’s CEO:

    ‘Our mission at BioCryst is to bring our therapy to any patient worldwide that needs it, regardless of their ability to pay.’

    The commitment, Charlie admitted, will take a few years to achieve, but it is central to BioCryst and drives them every day. The GLW audience responded to such a commitment with intense applause.

    Concluding, the BioCryst representatives told the audience that they looked forward to a brighter future for every HAE patient, young or old. After showing a short film of a young person with HAE, both agreed that personal stories: ‘Put extra fuel in our tank.’ BioCryst is just getting started helping more people in the HAE community.

  • Breakdown of the breakouts

    Nine regional breakout sessions across two hotels allowed every attending patient advocate to have the chance to discuss, brainstorm, and exchange knowledge. Global Perspectives was able to drop into each session and saw colorful balloons strewn across the floor in one session. In another, chairs were arranged into a circle to aid discussion. In a third, participants were hearing a presentation on the latest developments in their region. To share what happened in each session, Global Perspectives spoke to each RPA breakout lead and patient advocates from around the globe.

    RPAs and Member Organizations tell their story

    It was clear that no two breakout sessions were entirely the same. Whilst some key resources were shared with all participants, notably a new Patient Travel and Relocation Guide and a Virtual Meeting Resource, each RPA’s session was tailored to the needs and interests of their Member Organizations (MO).

    Central America and the Caribbean
    Javier Santana told Global Perspectives that each country discussed strategies and future plans. Participants offered their support to other countries, sharing resources, knowledge, and ideas to help others.

    From this breakout, we heard from Tarya Corrales from Costa Rica. Working with huge energy, HAE Costa Rica now supports more than 75 patients. Tarya is also HAEi’s youngest MO lead. Global Perspectives started by asking her what she took from the breakout session. She told us: ‘It was very helpful to listen to other countries’ advances and ideas on how to apply them in Costa Rica.’ Her reason for being at the GLW will likely resonate with many, she wanted to take action as a patient. Global Perspectives asked about the situation in Costa Rica. Tarya told us: ‘Costa Rica has made positive progress. There are two treatments available for patients with HAE.’ Finally, Tarya told us that she would like other people with HAE to know they are not alone. She said: ‘There are thousands of people with HAE who have been able to get ahead and be happy.’

    Across Asia
    In the Asia group, facilitated by Yong Hao Lim, there was a similar spirit of collaboration; however, the focus was on regional priorities such as patients’ mental health and how best to engage with pharmaceutical companies.

    Global Perspectives got the views of Makiko Matsuyama from HAE Japan. She referenced the fact there were similar challenges across Asia. She said: ‘The situation regarding HAE treatment in Japan is currently one of the more advanced in the world. We learned about the challenges facing other countries in the same Asian region, which are similar to those we faced many years ago, so I felt that sharing best practices would be helpful to other countries. We need to work together even more.’

    Both in the breakout sessions and in subsequent discussions, Makiko told us that everyone has similar challenges, but hearing how others have overcome them inspires them to action. In particular, HAE Japan was encouraged to proceed with an event for children based on colleagues’ successes.

    Finally, she said, stigma remains an issue in Japan, and one they are actively working to address by showing people living healthy lives, and that diagnosis and treatment can translate to a life free from attacks. You can learn more about HAE Japan’s work on stigma in ‘Cultural sensitivity and stigma reduction strategies’ as Makiko spoke during one of the Masterclasses on the main stage.

    Scandinavia and German-speaking Europe
    In the session focused on Scandinavian and the German-speaking nations in Europe (Germany, Austria, and Switzerland), Global Perspectives heard about the struggles for recognition of the disease in certain countries, something which was understood by all present, echoing the value of shared stories.

    Global Perspectives managed to catch some of the conversations in this group after dashing across from the other hotel. We heard participants from HAE Finland talk about their situation and knew it would be essential to get their input. Jenni Paasilinna kindly answered our questions. She and colleagues from Finland felt that the small, casual format of a breakout really aided conversation, and that conversation helped them to feel that they were not alone with the problems they faced in finding doctors capable of treating and diagnosing HAE. The breakout session, Jenni felt, also made it easier to ask questions about tools like HAiE FocalPoint and HAEi Connect.

    Jenni told us that in Finland, despite every University Hospital in Finland having one doctor who specializes in HAE, the level of knowledge can vary widely, with some knowing very little and being unable to diagnose or treat HAE properly. One doctor is nearing retirement, leaving patients concerned in case his replacement lacks the knowledge and experience he has.

    Jenni felt that the whole GLW was: ‘an amazing opportunity to connect with people from other countries with the same experiences as a patient and a caregiver.’

    America, Australasia, and South Africa
    Fiona Wardman led a large group of English-speaking MOs, from North America, South Africa, Australia, and New Zealand. One of the key topics was how to maximize engagement with the information and resources being developed, especially social media. Fiona told us that despite differences in MO sizes, the spirit of collaboration was strong, and everyone left inspired to drive greater impact.

    A big discussion point included making the most of content, such as videos, webinars, and online meetings. One idea was to create something that was ‘evergreen, ‘ meaning the topic would be endlessly relevant, such as ‘What causes HAE?’. Alternatively, creating a long video that could be broken up into bite-sized chunks and shared on social media also meant any investment of time or money would be well made.

    With such a large group, we heard from three participants. Janice Strydom (HAE South Africa), Olivia Worthington (HAE Australasia- New Zealand), and Lisa Facciolla (US HAEA.)

    Olivia told us that the breakout session was really productive. It was ‘fantastic,’ she said, to get in a room face-to-face with other HAE leaders. She said having an: ‘open discussion about the wins and challenges we face, especially in larger and smaller organizations, meant that learnings were shared. And now we’re all connected, it is easier to reach out for information and support.’

    ‘The breakout format was a real highlight of the 2024 HAEi Global Leadership Workshop. I took lots of notes and had a lot to take away from the session.’

    Janice from South Africa also felt the informal setting made it easier to brainstorm solutions and that her team had gone away with new ideas that they could explore further. It also allowed them to expand their network and share their own experiences.

    One of the most significant issues facing patients in New Zealand, according to Olivia, was the lack of modern prophylaxis. The small patient population and complex funding system made access a challenge. However, she was keen to stress that despite this, they felt that there was hope. Part of this, she said, was knowing the latest information about HAE and its treatment. This is the best way of getting the best care, she felt.

    For Janice, the situation in South Africa is characterized by low diagnosis rates, although an increase in healthcare professional knowledge and awareness has led to more frequent referrals. Like New Zealand, they have a therapy for acute attacks but lack modern prophylaxis.

    Janice wanted people with HAE all over the world not to see their condition as a disability. She told us that: ‘HAE can be challenging, but with the right support, patients can live normal lives.’ She quoted the famous American psychotherapist Dr. Ruth Westheimer, who said: ‘Our way is not soft grass; it’s a mountain path with lots of rocks, but it goes upwards, forward, towards the sun.’

    Lisa Facciolla was part of a strong contingent from the US HAEA team, present in the breakout session. She felt strongly that the breakout sessions represented an incredibly productive time where people exchanged ideas freely. Part of the value, she felt, was that by sharing ‘unique victories,’ whether an inspiring event or youth outreach, everyone grew stronger and more effective. Such sessions and the wider GLW were a crucial way to come together with others and share knowledge.

    ‘When we come together to share, truly great things happen. The breakout session was the perfect example of that.’

    Many advocates may look enviably at the medicines available to treat HAE in the US, and Lisa acknowledged that they are fortunate to have 8 approved therapies; the work of the US HAEA is far from over. She felt that they must continue to advocate to safeguard access, so their community can get medication and the reimbursement needed to afford them.

    Mediterranean, Central Europe and Middle East
    Maria Ferron and Michal Rutkowski worked in tandem, bringing together their groups to provide a dynamic platform for collaboration. Their sessions were structured according to key themes in their regions, with these topics generating a lot of passion and energy. Maria comments that the informal nature meant more networking was possible, leading to greater connections between individual advocates.

    What came out strongly from all of the session reports was the sense of togetherness and encouragement. Although every organization reported challenges, no one was alone in their issues, and everyone was ready with an idea or an experience that could help overcome the problem.

    Global Perspectives spoke to Peter Strahl from HAE Hungary. As a veteran of HAEi conferences, having attended since 2018, Peter said that the breakout session offers the chance to build their organization’s capabilities and hear about the work of other countries. Peter explained that one of the challenges facing HAE Hungary is trying to get more active participants in the MO. One of the ideas he shared was their plan to organize an HAE ‘club,’ in which they will update attendees on the latest news in HAE globally and in Hungary.

    Peter wanted to thank everyone who works hard to support HAE patients with what he called their ‘struggle,’ he said this effort has led to many achievements for people living with the condition.

    HAE patients in Morocco face many challenges, and it is a struggle to access necessary medications, according to Meryam Jennane, of HAE Morocco. She said that networking with pharmaceutical company representatives is critical to their success. Sitting down with fellow patient advocates was valuable, she felt, saying: ‘It gave us an opportunity to learn from the experiences of other organizations, which is essential as we continue to raise funds and identify new patients.’

    South America and Mexico
    Fernanda de Oliviera Martins led the South America and Mexico breakout session, in which she aimed to foster connections, learning, and fun. In a large group of around 70 people full of energy, there was a chance to see old friends and meet new people. There were quizzes to test knowledge, and, as Fernanda explained, the balloons were part of an activity to highlight shared problems and opportunities.

    After the session, Global Perspectives caught up with the team at HAE Venezuela. They said that the session was an excellent opportunity to network with the other organizations in Latin America and strengthen the support they receive. Despite having no available medication, HAE Venezuela remains committed to supporting patients and fighting for better, and they felt that the GLW provided a great opportunity to gain up-to-date knowledge and tools.

    Sub Sahara Africa
    Patricia Karani, the RPA for Sub Sahara Africa, focused her session on how to resolve challenges facing countries in the region. She said the main issues were access to HAE tests and lack of correct information amongst healthcare providers and patients.

    One of the participants in this breakout was Andrea Ntanga from HAE Mozambique. She said that connecting with representatives from countries like Kenya and Comoros was ‘a privilege’, as they faced challenges similar to those faced by Mozambique. In addition to the chance to co-create solutions, Andrea felt that cross-cultural dialogue was an essential part of the process.

    As a researcher in the biomedical field, Andrea enjoyed the chance to meet and discuss with healthcare professionals and patient advocates. In common with many in the global HAE community, Andrea wanted people with HAE to know they are not alone.

    ‘Every voice matters, and together we can drive change and improve access to care.’

    South Eastern Europe
    Global Perspectives learned a bit more about this session from Sofia Papalexiou of HAE Greece. Sofia told us that it helped to understand the common issues everyone faced, as well as just getting to know one another better. In common with many participants, Sofia felt that exchanging news and ideas was a real benefit.

    ‘It was helpful to meet with friends from all over the world with whom we share the same goal, and take inspiration and strength from each other.’

    As HAE Greece is a reasonably new MO, Sofia felt that they are trying to build trust with patients, through projects to improve quality of life. Ultimately, their goal is to get more people involved with their MO.

    Information and misinformation: A breakout session discussion

    A topic for discussion in the breakout sessions was how to engage patients in a safe, professional way. For some, that meant having both public and private Facebook communities, allowing a private space for community members to share experiences. Moderation challenges were discussed, as well as respecting peoples’ opinions while ensuring access to good quality evidence-based information.

    All of these conversations led Global Perspectives to ask: What are the most common and most worrying pieces of misinformation people in the community have heard about HAE?

    Before we begin, individual responses and experiences of people with HAE are always respected, and if sharing seems to help someone else, that’s fantastic. Global Perspectives always recommends checking any questions about any aspect of HAE with your HAE doctor/ healthcare professional.

    For many, foods seemed to cause a lot of misunderstandings. One suggestion was that eating chicken caused swells, whilst another participant shared that well-meaning relatives suggested that eating pork led to attacks. One participant mentioned a patient who noticed a difference after reducing sugar intake. The reason for this, others felt, could be as simple as losing weight, which can lower stress on the body leading to fewer attacks. Drinking more alkaline water was suggested to one participant by a friend. Individual responses and experiences of people with HAE are always respected, and if sharing seems to help someone else, that’s fantastic.

    The group agreed the desire to find a ‘reason’ may be driving misinformation. One participant told the story of their diagnosis; her family desperately logged all foods in an attempt to isolate a trigger when it turned out that stress led to the attacks.

    Other misinformation related to treatment. One person had been asked if HAE medicines are addictive. This is categorically not the case. When people are prescribed strong pain medication such as opioids during attacks, these types of medication can lead to addiction. Specific HAE medicines do not work in a way that can make people addicted.

    The success of prophylactic medicine meant that some patients had asked whether they might go into ‘remission’ after not having attacks for some time and perhaps they could stop their preventative treatment. It was important, a breakout participant said, to talk only about ‘attack-free.’ Right now, there is no cure for HAE. If someone is on prophylactic medication, controlled and not experiencing attacks, the medication is doing that.

    Overall, the group felt that misinformation could result from people who may be having a lot of attacks, clinging to anything as a source of hope. It can also be difficult, people felt, to counter such misinformation as there is nothing to disprove the assumption, which may make healthcare professionals reluctant to engage. All felt that this was an ongoing conversation and opportunity to share learnings.

    Perhaps you and your colleagues have experience of misinformation in HAE? How do you deal with it, and what tips do you want to share? You can contact Global Perspectives via news@haei.org to share your experiences and we’ll keep an eye on the conversation.

    A few tips on misinformation

    • Consider asking that no medical advice is shared on social media platforms to limit potential misinformation.
    • Consider if misinformation should be deleted or responded to with facts and evidence; perhaps inaccurate or misleading information can be addressed by a medical professional.
    • Moderation can be powerful, but takes time. A strong social media community will often self-moderate by challenging outlandish or inaccurate ideas.

  • Masterclasses in Building Stronger Patient Communities (1 of 2)

    ‘There was no sense of community. Everyone was struggling alone,’ said Marijk Beekman of HAE Netherlands, who was no doubt speaking for many when she told the GLW about the situation facing people with HAE.

    The second morning of the GLW continued the advocacy Masterclasses, hearing from RPAs and patient advocacy leaders. These Masterclasses tapped into the wealth of experience, ideas, and passion of the global HAE community focusing on the key challenges in setting up and running a successful MO. The first Masterclass, understandably, started at the beginning:

    Registering and managing a patient organization

    Director RPA Program, Michal Rutkowski welcomed to the stage Renata Martins Marchese from Abranghe Brazil (HAE Brazil) and Marijk Beekman of HAE Netherlands.

    A nervous Renata was given a supportive extra round of applause as she responded to a question about why she established an organization in Brazil. She started by telling the audience that there was an understandable personal desire to get treatment and have a better life, but swiftly it became about all the other people she saw that had the same disease.

    The critical first steps to an organization are writing the rules, including founding principles like the organization’s mission and goals, and registering with the appropriate authorities. Without this, Renata told the audience: ‘it is impossible to receive donations or have bank accounts.’ For much of this, a lawyer advised them to ensure the relevant Brazilian laws were followed.

    The Brazilian HAE organization now has around 1,600 members, but to begin with, Renata stressed that to get the support you need, just ask. Renata asked everyone and usually said: ‘Please help me. Do you know how to do this? Please help.’

    Moving onto finances and funding, Renata said that the pharmaceutical industry initially approached them as they worked to bring products to the Brazilian market many years ago. Since then, their finances have grown, but it is, Renata said, essential to ensure sustainability. HAE Brazil has an annual work plan in which they allocate budget to projects but not at the expense of fixed costs such as IT or meeting space.

    ‘A lot of patients didn’t even know what HAE was. We identified an enormous lack of information,’ said Marijk, when asked about the immediate needs of the HAE community in the Netherlands. To address this, Marijk partnered with healthcare professionals on the text of a new website. This was followed by an information day to bring patients together and show that it is possible to live your life without having attacks all the time. Subsequent programs have told patient stories, and specific activities for young people are being worked on.

    A specific issue for the Dutch organization was that it was for people with all forms of angioedema. Ultimately, Marijk said, the intention is to create an organization specifically for HAE, but this is a long-term goal.

    One tip from Marijk was to keep an eye on the HAEi website. ‘We always go there first to see if there’s something we can use, some resource or program that we can use.’ Another was the importance of proactivity. In common with Renata’s experience, Marijk said no one will come to you. Just keep reaching out, she told the audience. 10 no’s in response might become a yes on the 11th time.

    ‘If I struggle, I reach out to my Regional Patient Advocate, or I reach out to other people to ask if they know what to do, and perhaps we can do it together. We’re HAE patients, so we’re adaptable people.’

    Again, in line with Renata’s annual work plans, Marijk outlined how every year, the board of the HAE Netherlands sit down to review what they’ve achieved and gather data on the most critical priorities next year.

    One of those priorities was in-person meetings. According to Marijk, it was a big request from her community, so they had just completed their first such meeting. Due to its success a future meeting is planned, to which they will also invite patients from Belgium, where there are language similarities, helping to share knowledge.

    Michal’s final question was about bringing people into the organization. Marijk said that sharing information on social media, newsletters, and emails can help, but ultimately she said that it’s important that people see that they’ve missed something by not being involved.

    Her aim, she said, is to have people think: ‘Wow, they are a bunch of cool people. I want to be part of that!’

    Increasing patient group numbers

    ‘The mantra is: Every contact you make, every single one of them is a potential member.’

    Tony Castaldo, in his role at HAEA, was in conversation with Fernanda de Oliveira, the RPA for South America and Mexico. Their topic of discussion was how to attract new members to a patient advocacy organization.

    Now celebrating its 25th anniversary, the US HAEA has grown to over 10,000 members. When HAEA first started as a fully volunteer organization, everybody was aligned with the concept that there is incredible strength in numbers. Part of this came from the realization that pharmaceutical companies wanted to know how many people have a disease before investing and that you can achieve more when more people come together.

    Tony explained that US HAEA has the philosophy of the ‘big hand.’ He explained that: ‘This big, warm, happy hand has Velcro on it, you know the sticky stuff. Anytime anyone comes into our orbit with a swelling disorder, we want them to get stuck to that warm hand.’

    Practically he offered the following tips:

    • Have a very clear statement of purpose that everyone can see.
    • Reflect the needs of people in your country. People want to be part of something that meets their specific requirements. That will vary, depending on where you are in your advocacy journey
    • Make finding and activating new members a primary organizational goal.
    • Consider where folks with swelling disorder will be, such as hospitals or specialized centers
    • Every communication you make with anybody, make a pitch to sign them up
    • Be persistent and kind. Always keep that positive attitude
    • Always be on the lookout for opportunities to use your creativity
    • Show them that you’re a helping organization.

    Regarding the best tools to reach people, Tony mentioned the free web hosting service and other tools provided by HAEi. ‘Is there anybody that doesn’t like free’ he asked, light-heartedly.

    Beyond using the web, social channels, and, wherever possible in-person meetings, Tony highlighted the importance of good relationships with HAE specialist healthcare professionals. These are the people who are constantly in touch with people with HAE or unknown chronic swelling disorders.

    ‘Healthcare professionals are finding HAE patients. Make sure that every one of those are referred to your organization.’

    Cultural sensitivity and stigma reduction strategies

    ‘People don’t want to feel that they’re different,’ said Janice Strydom of HAE South Africa, encapsulating the issue of stigma around a rare genetic disease and the challenges it can cause to advocacy work.

    Janice Strydom and Makiko Matsuyama of HAE Japan spoke about stigma reduction and cultural sensitivity in the third GLW masterclass. Asking the questions, Fiona Wardman.

    Expanding on the problem of stigma, Janice mentioned that even when one patient is diagnosed, it can be hard to encourage the rest of the family to be tested, despite the dangers of undiagnosed disease. Some, she said, don’t feel they have regular enough attacks, but for some, it’s just not wanting to acknowledge that there’s anything wrong. They don’t want to stand out in society.

    Janice’s own experience in employment highlighted one stigma. A routine medical before starting a new job resulted in Janice sharing her HAE diagnosis. Providing the diagnosis meant she lost her job and led to persistent problems within the sector where she works. According to Janice, people hear about these kinds of experiences, and they stand in the way of people coming forward for testing.

    Makiko felt that stigma was a big topic in Japan. Her perspective was that for Asian people, there may be culturally less openness around an illness like HAE, and stigma was something she heard often from patients.

    Fiona asked both advocacy leaders what course of action they take when someone doesn’t want to be diagnosed. In South Africa, Janice said, it’s essential to show the benefits outweigh the risks. She admitted that, due to limited treatment options in the country, this can be difficult. However, there is power in putting people in touch with other patients so they can hear their experiences first-hand.

    Makiko agreed that telling your own story is powerful. She lost her husband due to HAE, but despite this, her message was a positive one. ‘Don’t be afraid. You can enjoy life,’ is what Makiko tells people.

    Fiona asked what other steps your organizations have taken to address barriers to seeking support and joining the HAE community.

    Janice stressed that there is a very diverse cultural community in South Africa, with 11 official languages, so there are many differences. However, lack of knowledge, fear, and the importance of the family unit were common themes for everyone. As a result, much of HAE South Africa’s information sharing focuses on reaching beyond the patient to their wider family.

    Makiko expanded on having an upbeat message. She said it was essential to show a positive attitude. When TV journalists ask if she is sad about the loss of her husband, she turns it around to something positive about what can be achieved.

    Working with a broader rare disease group in South Africa, Janice saw an initiative that aimed to reach traditional healers, who are often the first support for members of the community. HAE South Africa is now working with these traditional healers to ensure they understand HAE and can refer any patients to them.

    HAE Japan has engaged a genetic counselor to attend patient meetings and to talk to HAE patients, as this can help people understand the disease better.

    Summing up, the experiences shared in the masterclass made clear that:

    • The openness of shared stories helps understanding, especially if it shows there are no wrong questions
    • Regular communication helps. If you have experience with the media, use that. In some countries, such as South Africa, a simple WhatsApp group can be very successful.
    • Build collaborations. In Japan, there is a consortium of doctors, academics, patient advocates, pharmaceutical companies and employers to raise awareness

    Making the most of media and events

    Stepping next onto the big, big stage at the GLW were Michelle Cooper of HAE Canada and Danail Dimov of HAE Bulgaria. Natasa Anjeleska, RPA kept the conversation flowing.

    After agreeing that Bulgaria should go first, purely alphabetically, Danail was asked about his organization. He remarked that it started officially in 2018 and has ‘had a lot of fights’ in the years since. Their success, he said, has been such that there are many treatment options available, including prophylaxis, ‘which is something for a small country like Bulgaria, which is not very rich.’

    For Danail, his passion project was a recent documentary about HAE, shown on Bulgarian national TV. Inspired by the film Special Blood, they focused on Bulgarian stories and patients and agreed with presenters in the earlier session that sharing stories can help address stigma.

    In Canada, Michelle said they were proud of a recent awareness campaign that lit famous Canadian landmarks in HAE’s signature color. ‘But what color is HAE?’ Michelle asked. The answer came during the HAEi Regional Conference Americas, held in Panama: Purple. The campaign’s culmination was that the famous CN Tower in Ontario went purple during hae day :-).

    Michelle mentioned using the skills of patient members. One of their patients was a specialist in social media, and their plan took HAE Canada’s activities to another level.

    After the HAE documentary aired in Bulgaria, Danail was contacted by healthcare professionals, including an Associate Professor with two decades of experience who had never heard of HAE. As soon as they knew more, they identified two potential patients.

    Despite the challenges of getting TV media interested in the documentary, Danail knew that TV was the most used media in Bulgaria. Reacting to what she’d heard, Michelle felt inspired enough to try a documentary themselves, especially if it meant creating more long-term opportunities for awareness beyond a single moment or day.

  • Masterclasses in Building Stronger Patient Communities (2 of 2)

    Having an impact at the local and national level

    ‘What inspired me to get started in advocacy? I found my community. I found my people. We united for the same goal.’

    – Ersan Sevinç, HAE Türkiye

    Ersan Sevinç of HAE Türkiye and Carla Goachet Boulanger of HAE Peru discussed strategies to have an impact locally and nationally. Yong Hao Lim of HAEi performed double duty, acting as interviewer and translating Carla’s Spanish answers into English for the audience.

    Responding to the same question as Ersan, Carla felt the driver was the sheer lack of information on HAE in Peru. Thanks to a huge amount of hard work and partnerships with policymakers and fellow advocates, HAE is now included in the national list of rare diseases published by the Ministry of Health. The first clinical guidelines for treating HAE in Peru have also been produced.

    In Türkiye, Ersan indicated that their efforts have also been successful, with access to modern therapy and some medicines even produced domestically.

    Part of the success in Peru was down to partnerships and presence. HAE Peru representatives would travel to every region to hold talks and seminars with healthcare professionals. Now, there are governmental online platforms that allow them to reach even more doctors, especially as HAE Peru is a small organization with limited resources and is legally prohibited from seeking support and funding from the pharmaceutical industry.

    As a result, Carla told the audience they had to become creative about fundraising. They’ve had bingo and raffles amongst members, their family and friends. Additionally, you can persuade small businesses to offer money or direct support, such as refreshments at a meeting. It all helps.

    Ersan felt that different media offers opportunities, particularly in a large country like Türkiye. They discovered that one of their members was on the TV show, Master Chef. He spoke about his illness on live TV and his social media, which led directly to 10 new patients being diagnosed as they contacted HAE Türkiye after seeing his story.

    Carla and Ersan had many ideas for ways to have more impact. These include:

    • Develop a strategy with data, which you can share with decision-makers. Ersan said: ‘In the end, decision-makers only look for the numbers’
    • Offer a range of events. During COVID, a Turkish online event attracted more than 250 participants and lasted four times longer than expected due to the volume of questions being asked
    • Act as a team. Carla recommends working with at least one doctor as well as patients. Collaborating with other rare disease organizations can maximize impact.
    • Scale over time. The broader your reach, the greater the efforts needed to maintain it.

    ‘No matter what the obstacles, keep moving forward,’ Carla urged the audience.

    Getting political to get results

    Despite her young age, Tayra is a seasoned patient advocate and has been part of successful efforts in her native Costa Rica to secure funding and access to HAE medication. With his background in HAE advocacy and government affairs, Javier Santana, RPA, posed the questions in a masterclass dedicated to working with government and political individuals and groups.

    The first topic of discussion was the importance of collaboration with governments. Tayra told the audience that HAE is not seen as a disease in some countries; engagement is necessary to change the situation. She recalled that even after direct communications between HAE Costa Rica and the health authorities had started, several patients died due to lack of treatment. By the end of the process, patients in Costa Rica had approved medication, doctors who specialize in HAE, and an ID card that identifies them in the case of emergencies.

    Acknowledging that approaching government representatives can be intimidating, Tayra said these contacts are essential. Governments are the people who buy modern HAE drugs and ensure patients are treated with dignity and compassion. It is up to us to educate them, she said.

    ‘You elect government officials. They are there to help you.’

    One important point from the discussion was the transition between representatives after elections and the harm this can do to campaigning. Javier and Tayra both agreed that working to have HAE laws passed will ensure that even if politicians and governments change, people with HAE will still be protected.

    Javier offered his tips for what needs to be in any proposed law:

    • Include hae day :-) in the law to create awareness annually
    • Create official identification of HAE patients so that it is easy to know how many people need support and treatment
    • Include HAE on any list of catastrophic diseases (diseases that require prolonged hospitalizations or recovery)
    • Adoption of HAE treatment guidelines
    • Courses related to HAE should be included in medical school curriculums
    • The right to access HAE tests
    • The HAE specialist decides access to HAE medication
    • Plans to cover the cost of HAE treatment

    Tayra shared the following tips for actions to take to be successful:

    • Identify the needs of the patients in your country
    • Collaborate with people from HAEi who can support you
    • Make a list of all the Stakeholders who you need to meet; for example, health ministers
    • Find out who within your organization can help or might have prior experience or relationships with politicians
    • Work as a team. Success is more than one person’s job.

    Tayra explained that HAE Costa Rica owes a lot to her great colleague, Tiana Madrigal, who made multiple efforts to seek action from the Government. Tiana spoke to the media and secured the first meeting with the Minister of Health of Costa Rica, where she was clear about the needs of patients and the problems in hospitals. ‘It is up to us to maintain the great effort she and other colleagues made,’ Tayra told the audience.

    Pharmaceutical company collaborations

    Unfortunately, Ahmed Ali Abudahir, HAE Libya, could not attend the GLW due to travel issues, so Fiona Wardman, RPA, flew solo on-stage and shared her vast experience working with the pharmaceutical industry at the global and national level.

    She started by reminding the audience that HAEi Advocacy Academy offers free courses on working with the pharmaceutical industry, and these are a great first start for everyone. It provides information on what to do, what not to do, and how best to work with them, and it explains the rules and codes of conduct that pharmaceutical companies must adhere to.

    ‘There are lines that industry and advocacy organizations cannot cross. As advocates, we must be ethical and professional, as well as have open discussions.’

    Referencing her experience in HAE Australasia, Fiona described how they managed relationships with the industry. By discussing goals and objectives, she said it is possible to align on activities. Once that happens, a funding proposal is usually needed, which opens up different options. She suggested that an ‘unrestricted grant’ is the best way to go, as it provides more scope for how you can spend the money to deliver the purpose for which it was agreed upon.

    At this point in the process, Fiona said anyone with questions should contact their RPA, who can advise and support them. The first proposal might not be accepted, but there are always chances to revise and resubmit.

    Despite the source of the money, Fiona made it very clear that you must ensure you stay true to yourself and what you need.

    It is also vital, she said, to remain open to all, which can be difficult in countries with only one medication and company. An organization should never align so much with one company that it closes the door for others. HAEi remains strictly company and product-neutral to ensure there are options for everyone.

    Although Ahmed couldn’t be there, Fiona represented his voice by sharing his case study.

    The situation has been very challenging in Libya, but by working with a medical supply organization, Ahmed could streamline the procurement process and ensure medication was available when needed. The HAE Libya team needed to persistently lobby health authorities and the medical community to achieve this. Ahmed advised collaborating with medical supply organizations as part of a strong network, which can accelerate access to necessary treatments.

    Fiona added her advice to Ahmed’s. She suggested:

    • Consider privacy. Keep patient data to yourself and never give out patient names to the pharmaceutical industry
    • When you get funding, be careful to use it for the purpose intended
    • Be transparent and clear about where the money is going and how it has been used
    • Show the need for treatment to the pharmaceutical industry
    • Talk to whoever will listen
    • Ask about clinical trials, as these can increase access to therapy, even in resource-constrained countries
    • Remain highly professional in relationships with all stakeholders

    Innovations in fundraising

    The next masterclass was dedicated to fundraising. Maria Ferron, RPA, invited Natasha Jovanovska of HAE Macedonia onstage and asked about successful fundraising.

    Natasha described a children’s workshop that took place in a Macedonian park. The weather was good, and there were many activities for children, including making friendship bracelets. The biggest idea was for young people to experience trying activities while having a swelling attack, achieved by putting dough on children’s hands to mimic the swelling.

    In addition to communicating the nature of the illness, Natasha felt that this was successful as it tapped into people’s willingness to donate more when they or their children are having fun.

    HAE Macedonia specifically promoted this event to women and mothers. They used this approach for another event in a shopping center. Natasha believed that people are more likely to donate if they’ve already spent money, making this a good location. The event included dancing acts and was in partnership with the shopping center.

    Knowing your audience was something Natasha took very seriously. In their outreach to healthcare professionals, they knew that social media reach would have limited impact due to the lack of IT skills among the target audience. It was far better to create an in-person event, especially if it offered professional accreditation, as this is a requirement for doctors to continue to practice.

    When asked about working with donors, Natasha stressed the importance of hard work and consistency, as earning the sponsor’s trust is vital. Natasha felt finding a sponsor that aligns with your mission and values was important. In health, sponsors are primarily pharmaceutical companies, but some other companies invest in social responsibility, which can also be a funding source. Natasha advised that being proactive with pharmaceutical companies is vital, as is understanding what both parties want from any partnership.

    Natasha said another piece of advice, which might seem counterproductive, was to invest a little to make more money. She referenced a Macedonian saying in which you may have to give up smaller fish to catch a bigger one. Her example was their first fundraising event, a big concert in a city park. They had performers willing to play for free but needed to pay for a sound system and licenses. By investing their own money, they earned the trust of future sponsors and ultimately attracted media attention, leading to national TV coverage.

    In conclusion, Natasha shared a favorite story. The famous inventor Thomas Edison was said to have made 2000 attempts to make the lightbulb. Far from seeing these as failures, Edison reportedly said that he discovered 2000 ways not to do it and one way it worked.

    ‘Always take the positive spin; eventually, you’ll find your own light.’

    Make sure your events aren’t a zoo

    Sadly, illness had reduced this masterclass from three participants down to two. Karina Langsager, one of the HAEi specialists dealing with event management, could not attend due to illness. Everyone wished her to get well soon. Patricia Karani, RPA, moderated discussions.

    She and Michal Rutkowski, HAE Poland, identified two HAEi tools designed to make event organizing easier and faster: EventZoo Lite and EventZoo Max. But what’s the difference? Michal was asked. He replied that it is simply a question of scale and complexity.

    EventZoo Lite
    It is a registration system for meetings under 100 participants, with no fees, flights, or accommodation; you are just registering attendees. It is, Michal said, very, very easy. You let Karina at HAEi know what you need at least 15 days in advance, and everything will be set up for you.

    From his experience of a recent youngsters’ event, Michal felt that EventZoo Lite made this easy. Ensuring all the data is captured in one place makes contacting attendees easy. ‘It also saves our time,’ he added.

    Patricia wondered about the level of customization offered. Michal said there is a wide variety of templates and custom options.

    EventZoo Max
    It is a system for events on a larger scale: over 100 participants. It also offers functionality to manage flights and accommodation, etc. Most people present will be aware of EventZoo Max, Michal said, as HAEi used it to manage and register participants for the GLW. Like EventZoo Lite, you can completely customize EventZoo Max.

    Patricia voiced the questions many would be asking: With so much personal information, how secure are these tools? Michal understood the concern and reassured everyone that these systems are designed to be highly safe, with privacy built in. Michal asked one of the system’s designers, IT expert Morten, about EventZoo security. Morten said the only chance of getting into the server would be to kidnap him. ‘And Morten looks like a Viking-it’s impossible to kidnap him,’ Michal quipped. The systems are very, very secure.

    To finish, Patricia asked Michal to summarise the benefits he had seen using these tools in HAE Poland. Michal listed the following benefits:

    • It makes the event a lot easier to organize
    • You have way less stress
    • You are in control of participant details and timings, such as arrivals or dietary requirements
    • It’s is GDPR compliant, so very secure with personal data
    • You can focus on what’s important: having a great meeting!

    ‘EventZoo is a state-of-the-art system. It’s free. Use this tool to make organizing your events easier.’

    – Patricia Karani, RPA

  • A flavor of the ACARE Global Angioedema Forum

    The ACARE Global Angioedema Forum 2024, affectionately known as the GAF, welcomed 250 healthcare professionals and pharmaceutical industry representatives from across the globe to a two-day celebration of the science behind angioedema and its treatment, with the meeting featuring a particular focus on HAE.

    The meeting was the first GAF from the ACARE network, an initiative by GA²LEN and HAE International (HAEi). The GAF ran parallel to the 2024 HAEi Global Leadership Workshop (GLW), and on Saturday afternoon, both meetings joined for a unique opportunity to share knowledge and network.

    Due to the breadth of the program for the GAF, Global Perspectives can only hope to give a snapshot of the scientific exchange. We bring you a flavor of GAF.

    The GAF in numbers

    With 250 attendees and 85 submitted abstracts, the GAF has already attracted some big numbers. However, that’s just the start. Here’s some more of the GAF in numbers:

    • 15 chairs
    • 28 speakers
    • 10 topics talks
    • 1 expert panel of 6 doctors
    • 4 poster walks and 8 poster walkers
    • 3 lunch workshops
    • 2 keynote presentations
    • 1 interactive patient journey session
    • 1 angioedema jeopardy quiz to test clinicians’ knowledge

    Opening and introductions

    Following the tragic loss of Professor Marcus Maurer, Professor Markus Magerl took on the role of opening the GAF. Fresh from his eulogy to his friend and colleague, Prof. Magerl opened the GAF by welcoming his peers, colleagues, and friends to the meeting.

    He noted that the European Accreditation Council for Continuing Medical Education (EACCME) had awarded the GAF 10 European CME credits due to the quality of the agenda, speakers, and scientific content.

    Prof. Magerl turned first to the ACARE (Angioedema Centers of Reference and Excellence) network as the driving force behind improved patient care in angioedema, and core to the development of the GAF. He told the audience that the network had reached 103 centers spanning 40 countries, and is now present in all inhabited continents.

    Next, Prof. Magerl drew the audience’s attention to the submitted abstracts. He announced that 85 abstracts were submitted. Sixteen were accepted for oral presentation and 73 for poster presentation. He said reviewing such a large body of scientific data is a big job, and Prof. Magerl thanked the entire GAF Steering Committee for their hard work and dedication.

    Are you looking for scientific data from GAF?

    If you want to read more about the wealth of scientific data presented at the GAF, 80 abstracts are available online.

    >> Just click here to access

    Prof. Magerl, on behalf of the GAF Scientific Committee, announced that the Young Researcher/Investigator award was given to Dr. Carolina Vera Ayala from the German ACARE at the Institute for Allergology, Charité – Universitätsmedizin Berlin. The Scientific Committee grants the award to an abstract they deem to be of the highest quality and authored by – either solely or as part of a team – a young investigator or researcher. Dr. Vera Ayala’s research was into stopping androgens in patients with HAE: “First Insights from the Global ACARE SHAERPA Study on Androgen Discontinuation in HAE Patients, an Interim Analysis.”

    Following the award, Global Perspectives asked Dr. Vera Ayala about her feelings on winning and the GAF in general. She said, ‘It was an incredible honor and a truly surprising and humbling experience. Hearing that my work was recognized with such a prestigious award filled me with immense joy and gratitude. It felt like a validation of the hard work and dedication that went and is still going into the project. I am deeply thankful to everyone who supported me, especially Professor Marcus Maurer, who got me involved in this great project and gave great advice. It is a moment I will cherish and one that motivates me to continue striving for excellence.’

    Global Perspectives also asked her about the GAF overall. She said, ‘Attending the first Global Angioedema Forum was an inspiring and enriching experience. It provided a unique opportunity to connect with professionals from diverse countries, exchange ideas, and gain new perspectives. The collaborative environment fostered meaningful discussions on global issues, and it was exciting to see different cultures and expertise come together to address common challenges in managing HAE. Overall, the event was not just about networking but also about learning, innovation, and building relationships that can lead to future collaborations and help improve the lives of our HAE patients.’

    ‘The GAF provided a unique opportunity to connect with professionals from diverse countries, exchange ideas, and gain new perspectives. Overall, the event was not just about networking but also about learning, innovation, and building relationships that can lead to future collaborations and help improve the lives of our HAE patients.’

    – Dr. Carolina Vera Ayala, ACARE Global Angioedema Forum 2024 Young Researcher Award Recipient

    First insights from the global ACARE SHAERPA study on androgen discontinuation in hae patients, an interim analysis

    Carolina Vera Ayala (Germany)

    Dr. Vera Ayala’s abstract was selected as an oral presentation, and she gave it on the first day. She told the audience that the research aimed to determine why, how, and with what consequences androgen treatment is stopped in patients with HAE, and to eventually lead to guidelines on how to discontinue androgen treatment and change to available new medication safely.

    The work consisted of research amongst 50 patients in six different centers, and the team intended to extend this to 500 patients.

    The key results, presented by Dr. Vera Ayala, were:

    • The mean duration of treatment in patients receiving androgens is 116.5 months
    • The most commonly used androgen is danazol
    • The most common reasons for stopping androgen treatment were side effects and fear of side effects
    • There was an almost equal split between physicians who favored tapering the dose of androgens before stopping and those who preferred to stop treatment immediately

    Dr. Vera Ayala called upon audience members to consider participating in the forthcoming large-scale global research phase. This data will be used to produce guidelines for patients and physicians on stopping treatment with androgens.

    Keynote 1: The Past, Present And Future Of Angioedema

    The session Chairs welcomed the first two keynote speakers on stage: Dr. Anjur Jindal from the Postgraduate Institute of Medical Education and Research in Chandigarh, India; and Dr. Henry Li from the Institute for Asthma and Allergy in Maryland, United States.

    Dr Jindal spoke first. In a wide-ranging presentation, he covered the following topics:

    • Epidemiology of angioedema
    • Classification and nomenclature of angioedema (DANCE)
    • Disparity in access to diagnostics
    • HAE with normal C1 inhibitor
    • Biomarkers for angioedema
    • Genetics of HAE

    Looking at the epidemiology in the Asia-Pacific region, he indicated widespread differences in prevalence, from 0.33 and 1.00 per 100,000 in Singapore and New Zealand, respectively, to as low as 0.02 per 100,000 in India.

    On access to diagnostics, he referenced a recent paper that showed that in Asia-Pacific, 71% of centers had facilities to test C1-inhibitor level, and 64% had access to C1-inhibitor function testing. He indicated that variations in access to testing had implications for the diagnostic approach. In some cases, the sensitivity of a C4 test can be increased by drawing blood during an emerging attack, but this does not improve the ability of the test to confirm if a person doesn’t have HAE.

    He also noted that HAE with normal C1 inhibitor can only currently be diagnosed by genetic testing.

    Looking to the future, Dr. Jindal suggested that there will be:

    • More collaborations between clinicians
    • Greater understanding of all types of angioedema
    • New ways of spotting and differentiating between various types of angioedema and predicting the severity of acute attacks
    • A point-of-care diagnostic test for HAE
    • Disease-specific patient-reported outcome measures specific to the needs of children and young people
    • More equality in access to diagnostic testing, especially in Asia-Pacific

    In conclusion, Dr. Jindal told the audience, ‘We need to use our experience in our experiments to achieve our expectations.’

    Following on from Dr. Jindal, Dr. Li focused on the treatment aspects. He outlined the marked changes and improvements to the classification and treatment of a wide range of angioedemas.

    Looking at the future of HAE therapeutics, Dr. Li highlighted that there are now sixteen existing or forthcoming treatment options that target six separate elements of the system in the body that lead to HAE.

    In conclusion, Dr. Li suggested, in common with Dr. Jindal, that there will be an increasing understanding of angioedema, leading to better diagnosis and classification. He was also upbeat about the future of treatment, with improved efficacy, quality of life, and easier administration all on the horizon.

    Poster walking

    In addition to a full agenda in the main auditorium, the GAF also hosted a packed poster room. While all the posters were deemed important by the Chairs and Scientific Committee, which of the 80 papers should you focus on if you were short of time? Poster Walkers were the answer. These senior clinical leaders curated guided tours of the available data to time-poor attendees.

    The poster walkers selected one poster per walk as the highlight of their walk. We offer our congratulations to the following presenters:

    • Impact of Hereditary Angioedema Attacks on Quality of Life and Ability to Work Among UK Patients Receiving Long-term Prophylaxis or On-demand Treatment Only. Patrick Yong, United Kingdom
    • Self-Reported Treatment Preferences of Patients Switching from Prior Prophylactic Therapies to Donidalorsen for the Treatment of Hereditary Angioedema: Results from the Phase 3 OASISplus Study. Danny M. Cohn, Netherlands
    • Predicting Hereditary Angioedema Prognosis with a Next-Generation Sequencing Angioedema Panel. Joseph Chiao, USA
    • Prevalence of Autoimmune Diseases in Iranian Patients with Hereditary Angioedema. Mohammad Reza Fazlollahi, Iran

    Here, Global Perspectives catches up with three of the GAF Poster Walkers to get their views on some of the key aspects of angioedema science that caught their eye.

    Dr. Philip Li, University of Hong Kong, Hong Kong
    What, if any, were the key themes from the posters you chose for your walk?
    There was a wide and good variety of posters on our walk. The themes were mainly on improving diagnosis and novel strategies for HAE management.

    Why did you choose the posters you chose?
    Based on novelty and equity to give representation from all regions, not just Western or industry-sponsored abstracts.

    What did you think of the quality and breadth of posters?
    Overall, very good and extremely broad, from developments in middle/lower income countries through to ground-breaking and cutting-edge technologies from developed regions.

    What would you want patients to know?
    GAF is a showcase demonstrating the global effort and collaboration (East and West) to tackle HAE together.

    What does the future of research in angioedema and HAE look like to you?
    We are no longer counting attacks or measuring symptoms; we are looking at quality of life – and, more importantly, normalization of quality of life. With that – we are looking closer and closer to a cure!

    Dr. Mark Scarupa, Institute for Asthma and Allergy, Maryland, United States
    What, if any, were the key themes from the posters you chose for your walk?
    I don’t think there was a specific theme. I was impressed by the diversity. There was translational research, multicenter clinical trial data, epidemiology, and posters on the impact of patient advocacy. This diversity was in the posters we selected and throughout the poster session.

    Why did you choose the posters you chose?
    I was most interested in a mix of trial results, as those posters speak to where HAE therapies are heading and epidemiology.

    What did you think of the quality and breadth of posters?
    Very high. Whether it was posters organized by HAE societies and patient-centered groups or scientists, they were well thought out and informative.

    What would you want patients to know?
    Patients should know the breadth of international interest and expertise in HAE. There are great minds and enthusiastic patient advocates present on all continents.

    What does the future of research in angioedema and HAE look like to you?
    Historically, we have focused on attack rate reduction, speed to symptom resolution, and mechanism of action. As therapies continue to evolve, there is increasing focus on patient quality of life and a hope that patients, over time, can view their disease as completely manageable.

    Professor Konrad Bork, University Medical Center, Mainz, Germany
    What, if any, were the key themes from the posters you chose for your walk?
    Together with Dr. Luis Felipe Ensina from Brazil, we reviewed the abstracts of 16 posters, which covered various topics around hereditary and acquired angioedema.

    Why did you choose the posters you chose?
    Dr. Ensina and I selected five especially outstanding posters based on excellence in content and presentation, and all posters led to a lively discussion.

    What did you think of the quality and breadth of posters?
    All posters were of high quality and were interesting for physicians, patients, and generally all people dealing with hereditary and acquired angioedema. Finally, according to the criteria mentioned, we chose one poster as the highlight.

    Keynote 2: The emerging spectrum of hereditary angioedema and the algorithmic diagnostic workup

    On the second day’s morning, delegates heard the second keynote lecture, this time on diagnosing the spectrum of HAE. The speakers here were Dr. Laurence Bouillet from the French National Reference Center for Angioedema at the Grenoble University Hospital, France; and Dr. Avner Reshef from the Angioedema Research Center at Barziliai University Medical Center, Israel.

    Speaking first, Dr. Bouillet concentrated on an algorithm-based diagnostic process for isolated angioedema. The plan focused on five aspects of diagnostic decision-making:

    • Definition of isolated recurrent angioedema
    • Drugs and angioedema
    • Family history of angioedema
    • Biological analyses
    • Response to therapy

    Dr. Bouillet discussed aspects to consider with recurrent angioedema, such as the sudden onset and long duration of symptoms, and that it resolves without damage to the body. The absence of hives is also important, as are reactions to drugs such as ACE inhibitors or some painkillers (NSAIDs).

    Dr. Bouillet also discussed the importance of family history in diagnosis. However, she cautioned clinicians to consider that 25% of mutations are de novo, meaning there is no family history, and that in 15% of people with a mutation leading to C1-inhibitor deficiency, there are no symptoms.

    Dr. Bouillet was clear that with any type of bradykinin-mediated angioedema, only a test of C1-inhibitor levels can rule out HAE due to C1-inhibitor deficiency.

    In conclusion, Dr. Bouillet outlined five key considerations:

    • Rule out drug-induced angioedema
    • Check out the family context with caution
    • Check out C1-inhibitor function and propose genetic tests
    • Assess the response to long-term prophylaxis treatment (anti-H1 and omalizumab)
    • Think about trigger factors and abdominal attacks

    Closing this keynote was Dr. Avner Reshef. He focused on the emerging spectrum of angioedema and its classification.

    Starting with the system of classification whereby angioedema was defined according to whether it was histamine-mediated or bradykinin-mediated, Dr. Reshef then indicated that around ten years ago, this moved on to a focus on known biomarkers and genetic mutations at the time.

    Fast forwarding to 2024, Dr. Reshef showcased DANCE, a new global consensus on how angioedema is defined and classified. He indicated the six key principles of DANCE:

    • A comprehensive classification covering all angioedema syndromes due to vascular-endothelium hyperpermeability
    • Integration of phenotypes, pathomechanisms, and genomics (types, endotypes)
    • Re-classification of ‘normal C1-INH’ genetic variants
    • Clear terms and consistent wording of acronyms/abbreviations
    • Agreed and consented by an international panel of angioedema experts
    • Assimilation of this new vocabulary into future scientific publications

    Turning his attention to the genetic work-up of HAE with normal C1 inhibitor (HAE-nC1INH), Dr. Reshef highlighted the following key considerations:

    • Patients who are suspected to have HAE-nC1INH should be assessed for known mutations underlying HAE-nC1INH
    • Variable family history and lack of specific biomarkers hamper the correct diagnosis and management of HAE-nC1INH patients
    • Genetic evaluation is an imperative unmet need for angioedema patients without a definitive diagnosis
    • Genetic testing is required to establish the diagnosis

    Dr. Reshef concluded by referencing that the DANCE work was published just days after the disappearance of Prof. Marcus Maurer and that he would continue to work with others to fulfill Prof. Maurer’s legacy.

    Lunch workshops: The unknown variant and what to do next

    During the lunch break of the GLW, Global Perspectives jumped into the GAF to bring readers a flavor of one of the 3 GAF lunch workshops: What to do when genetic testing shows an unknown variant? Hopefully, it will dispel some of the complexity around how and when genetic testing is used, and give patients more understanding of the challenges facing doctors trying to diagnose extremely rare forms of HAE.

    Professor Anastasios Gemenis from the University of Thessaly in Greece and Dr. Anna Valerieva from the University of Sofia in Bulgaria, led the workshop.

    Dr. Valerieva explained that the workshop aimed to help clinical colleagues understand what to do when there is difficulty in interpreting genetic test results and the mutations identified. Firstly, she reminded the audience most HAE is associated with a deficiency of the C1 inhibitor protein. For these people, there is ordinarily no need for genetic testing to diagnose. Complement tests that assess the function of the C1 inhibitor protein and C4 protein level are commonly used and confirm most cases of HAE due to C1 inhibitor deficiency. There are some cases when genetic testing may be used, such as when complement testing is inconclusive or in newborns whose complement system is not mature.

    Understanding the genetic mutation is important for diagnosis and treatment in the much less frequent cases of HAE where patients have normal C1 inhibitor values. You can learn more about this in Prof. Marc Riedl’s GLW presentation, ‘The latest on HAE with normal C1-inhibitor’.

    Dr. Valerieva indicated that eight mutations have been identified as leading to HAE with normal C1. However, this doesn’t account for all cases. The majority are still classified as unknown HAE.

    Dr. Valerieva helped explain some of the jargon surrounding genetic mutations:

    • De Novo mutations are those found for the first time in a family or a patient but have previously been seen in other patients.
    • Novel mutations are those which have never been reported in any patient.

    The clinicians then went on to discuss two cases. The first was a boy with HAE with C1 inhibitor deficiency. Would his younger sibling (a sister) be at risk of HAE?

    Genetic testing demonstrated a novel variant, a new mutation never seen before. However, Dr. Valerieva said it was not straightforward to interpret whether this causes disease. There is no previous data on such completely new mutations. Looking at family history can help, and bioinformatics can use computer power to analyze whether a gene or the resulting protein is likely to cause disease.

    Dr Valerieva said the resulting information should always be shared with the scientific community, as it will create a database that doctors can search to diagnose or give peace of mind to families.

    Through extensive genetic detective work, they determined the girl did not have HAE.

    The second case was related to a man suspected of having ACE inhibitor-related angioedema, but despite stopping these medicines over a year ago, he continued to have attacks.

    The assembled clinicians discussed medical treatment for the patient, as specific therapies act to diagnose the form of angioedema when effective. In this case, this course of action did not help. Ultimately, the treating clinicians needed not to discount HAE with normal C1. The resulting genetic testing indicated that they were right; the patient suffered from HAE associated with a plasminogen mutation.

    Dr. Valerieva concluded that family counseling can be challenging in patients with HAE with normal C1, as the mutation is only sometimes passed on to other generations.

    A spot of Angioedema Jeopardy

    While many patients and caregivers will be aware of their form of angioedema jeopardy relating to diagnosis, treatment, attacks, and quality of life, we invited clinicians to use the Angioedema Jeopardy section of the GAF. This session pits clinicians’ knowledge and skills against their peers in a series of questions with varying difficulty.

    Led by Professor Marc Riedl, who took the quizmaster role with aplomb, the audience split into two groups. Each audience member had a red card (representing false) and a green card (representing true). Replicating the Jeopardy TV gameshow board, Prof. Riedl offered each group a series of categories and points. Once selected, the audience was shown a statement, to which each member of the playing group showed their red or green card. The resulting average answer decided if the group was correct and took the points. If yes, the group selected another category and points; if not, the play moved to the other side of the audience.

    The expert panel on-stage offered their feedback on answers. Some questions caused a lot of discussion! One question that stumped much of the audience was about the make-up and role of ACARE. Less than half understood that the ACARE network is a partnership between GA2LEN and HAEi, compared to, e.g., UCARE, which is only a GA2LEN initiative.

    Eventually, at the end of a hard-fought game, one side of the audience won by only 10 points! One lucky winning team member even went home with an ACARE hoodie as a prize.

    The immediate feedback from the meeting was excellent and will be collated and shared in future Global Perspectives. Speaking on behalf of the entire GAF organizing team, Debs Corcoran, HAEi’s Chief Scientific Officer, said, ‘We’re delighted with how wonderfully this first GAF was received and the knowledge and energy participants are taking home. Thank you to everyone who made the meeting a success!’

  • HAEi and ACARE: A strong partnership

    For the Saturday afternoon sessions, participants in the GLW were joined by their healthcare professional colleagues from the GAF to create another mega meeting, “GAF Meets GLW,” of around 750 people.

    It was fitting, therefore, that the first session was a presentation on ACARE, a joint initiative bringing together the patient advocates at HAEi, and the expert physicians in GAL2EN (an organization dedicated to allergy and asthma research). Tony, Henrik, and Professor Markus Magerl took the stage to provide an update.

    Tony and Henrik opened the conversation by taking everyone back to 2019 and heady, pre-COVID days. They told us that the HAEi board wanted to enhance the relationship between physicians and patients, but they just couldn’t think of how to make it happen. Scrabbling around for a solution, Tony and Henrik got their inspiration from a mention of Professor Maurer’s name. He’d pioneered a network of clinics in urticaria; why couldn’t this be repeated in angioedema? Well, it could, and the result is ACARE.

    ACARE has grown to over 100 member centers in more than 40 countries. The later Professor Maurer drove ACARE, but this work will continue.

    ‘ACARE is a key part of the quest to dramatically increase HAE patient quality of life.’

    – Tony Castaldo, CEO HAEi

    Referencing delays of five to ten years (or more) before an HAE diagnosis, Prof. Magerl said the common problem was one of education. Doctors don’t know how to diagnose HAE. However, even after a diagnosis, it is not self-evident that patients will get the correct treatment.

    At the heart of ACARE are programs that educate doctors. These include webinars, which in the last year have reached thousands of physicians from over 50 countries. Beyond webinars, experts travel globally to educate their clinical colleagues about HAE. In the previous year, face-to-face events have occurred in India, South Korea, and the United Arab Emirates.

    ‘How do patients benefit from ACARE? Put simply, it means better-trained physicians.’

    – Prof. Markus Magerl

    Educated physicians are the first step to better care. Every clinician is invited to have their center or clinic become ACARE accredited. The process involves a rigorous, 32-criteria audit to ensure they have the skills and the commitment to treat HAE patients well. Just a few of the criteria are:

    • Cooperation with colleagues in dermatology, gastroenterology, etc
    • Use of patient-related outcomes
    • Partnership with local or regional angioedema organization
    • Participation in research to extend access to new and forthcoming medication

    Prof. Magerl said that ACARE, along with HAEi, is also critical to the development of angioedema guidelines. The next guideline will be due in the next few years, and it will be essential to clarify how HAE should be effectively managed.

    ‘Caring for HAE patients is more than just taking bloods, making a diagnosis and writing out a prescription.’

    – Prof. Markus Magerl

    Finally, Prof. Magerl urged everyone to remember the ACARE registry. This may sound dull, he said, but without gathering together data on outcomes and side effects from as many people with HAE as possible, there is a chance rare symptoms, side effects, or long-term changes may be missed.

    ‘If you want to find a side effect that occurs in one out of 100 patients, you will have a problem with data from just 90 patients’, he told the audience.

    Tony, Henrik, and Prof. Magerl handed the stage to Fiona Wardman, Sarah Foltz Smith, HAE Spain (AEDAF), and Dr. Teresa Caballero to discuss the ACARE collaboration real-life benefits for clinicians and patient advocates.

    Collaboration between physicians and patient advocates: An ACARE example

    ‘Sometimes physicians have ideas about what the patient needs that are far from the reality,’ said Dr. Caballero, a Spanish clinician with an ACARE-accredited clinic. Dr. Caballero stressed the importance of gaining first-hand knowledge of patient needs and one of the many benefits she has seen from her close collaboration with AEDAF.

    Fiona asked what the benefits were for an MO. Sarah from AEDAF said that it’s just not sensible to work separately. Doctors can help set up a patient organization, identify patients, and encourage participation. She added that Doctors can give expert advice on aspects of diagnosis and treatment.

    ‘How does collaboration work then?’ Fiona asked. Both Sarah and Dr. Caballero stressed the need for shared and sustained communication. It is something that requires work on both sides. It is good to work together and know each other’s agenda.

    Asked about some of the fruits of this long-standing partnership, Sarah referenced a patient guide about treatment, trips, diagnosis, and many other aspects of life with HAE. Other programs have included patient and physician surveys to identify access to modern medications in Spain.

    In conclusion, Dr Caballero was asked what ACAREs can do for advocacy efforts, and she felt that the provision of scientific advice and input into drug funding applications would be a significant source of support.

  • Two tools to get ahead of HAE

    Taking advantage of healthcare professionals and patients being in the same room, Henrik Balle Boysen and Jørn Schultz Boysen grabbed ten minutes each to share the latest updates on two HAEi technological tools.

    Giving the fuller picture – Using HAE TrackR

    Henrik spoke first, reminding the audience that HAE TrackR is an easy-to-use electronic diary, and a personalized tool to empower every patient to take control of their HAE.

    He noted that more than 2,000 people have downloaded HAE TrackR since its launch, and this can be done from both Google Play and Apple App Store. And with 36 languages supported, it is available to many people.

    Henrik told the audience that fellow patients developed HAE TrackR, but it is also a tool for physicians as it promotes great communication between doctors and patients. When patients can share their HAE story, such as attacks, treatments, etc., it allows for more informed discussions and truly individualized care.

    Henrik outlined the next updates for HAE TrackR, which are due for release later in autumn:

    • In-app notifications as an alternative to text message reminders
    • Reminders about treatment use during attacks
    • Fast searching for batch and medicine numbering
    • Adding the Angioedema Control Test so that physicians can see how well HAE is managed
    • Integration of data into registries (where patients have approved)

    Henrik concluded by telling the audience that HAEi continued to act on the feedback received about HAE TrackR to make it easier and better to work with.

    >> Read more about HAE TrackR on haetrackr.org

    Your HAE companion

    ‘Ladies and gentlemen, HAE Companion is a life-saver.’

    – Jørn Schultz-Boysen, Executive Vice President Global Operations and Chief Compliance Officer, HAEi

    A pocket-size travel partner, HAE Companion, is developed to make travel and life easier and safer for people with HAE, said Jørn. At the heart of HAE Companion are emergency cards that can be downloaded to a phone, similar to a plane or train ticket. These cards provide essential details on HAE and sources of treatment and advice across 89 countries worldwide, with more to come.

    Information includes links to the MO in that country, ACARE centers, nearby hospitals, and doctors. Integration with mapping apps means it will even give you directions on how to get there, which is handy in an emergency, Jørn suggested.

    Jørn encouraged everyone present to have HAE Companion on their smartphone. Better to have it and not need it, than need it and not have it, as the old saying goes.

  • The latest on HAE with normal C1-inhibitor

    Making the complicated simple is a specialty for Professor Marc Riedl, but even he had to admit that HAE with normal C1 is difficult. He told the packed conference that ‘HAE with normal C1 (HAE-nC1INH) is a hot topic because it’s difficult.’

    Nevertheless, he accepted the challenge of explaining the progress made to date in understanding, diagnosing, and treating HAE-nC1INH.

    Starting with all angioedema, Prof. Riedl could quickly describe and categorize non-allergic angioedemas, of which HAE is one. If these angioedemas ‘hit the airway,’ he said, they’re much more dangerous. They can be highly debilitating. Prof. Riedl noted that we understand HAE Type I, Type II, and acquired angioedema pretty well. Using a traffic light system, he classified these forms of HAE with a green ‘go’ light.

    The traffic light changed to yellow ‘caution’ regarding HAE-nC1INH. We proceed cautiously, Prof. Riedl told the audience, ‘We understand a little bit, but there’s still lots of questions.’

    Finally, with HAE unknown (sometimes called idiopathic angioedema), Prof. Riedl said the traffic light goes red. ‘We don’t understand this condition well at all. Sometimes we don’t even know what’s the underlying cause’.

    Very importantly, Prof. Riedl stressed the need to ask specific questions. HAE is known to last longer and doesn’t have hives or itching. There can be specific triggers such as medicines, stress, or trauma. Also, the patient should be asked if allergy medicines work at all. And especially with HAE-nC1INH, familial and genetic elements are important. Prof. Riedl said all this and more helps guide clinicians to a diagnosis. Prof. Riedl stressed that after decades of study, we know HAE is caused by missing or ineffective C1 inhibitor, leading to too much bradykinin, making blood vessels leak and causing swelling. The test rightly, therefore, focuses on levels of C1 and C4 as these can reliably diagnose this HAE.

    Around the turn of the millennium, any confidence clinicians felt about their knowledge of HAE was shaken. Families were identified as having all the symptoms of HAE, but the tests showed normal levels of C1 inhibitor. ‘These people weren’t following the rules,’ Prof. Riedl quipped.

    Quoting the famous American writer Mark Twain, Prof. Riedl said: ‘It ain’t what you don’t know that gets you into trouble. It’s what you know for sure that just ain’t so.’ There is so much unknown or unclear that it is important to proceed thoughtfully.

    ‘It ain’t what you don’t know that gets you into trouble. It’s what you know for sure that just ain’t so.’

    – Mark Twain

    Complicated genetic testing has identified eight genetic mutations that cause HAE-nC1INH, but these versions of HAE often appear with troublingly different symptoms. As Prof. Riedl put it, ‘People with HAE don’t get hives. Well, now there’s this form; maybe they get hives.’ One of the significant questions is how many people have HAE-nC1INH. In one study, Prof. Riedl highlighted that HAE-nC1INH accounted for 20% of the people studied, so it is rare, but as he put it: ‘it’s not minuscule.’

    Returning to patient care, Prof. Riedl made it clear that when he’s sitting with a patient or a family with angioedema, he’s got to help them. Understanding cause and genetics is ultimately vital, but right now, what can be done to manage the condition?

    Sadly, Prof. Riedl mentioned that there has only been one controlled clinical trial, which hasn’t been published yet. All the available evidence is reports from centers or something that worked well for one patient or a group. There is some data on the benefits of tranexamic acid, Prof. Riedl suggested, along with medications like landelumab, berotralstat, icatibant, etc.

    ‘So how do I manage it?’ Prof. Riedl asked rhetorically. There’s no substitute for sitting down with the patient and their family and asking about the symptoms. Questions might include:

    • How many times do you have attacks?
    • What are those attacks like?
    • How long do they last?
    • Do you get hives or itching?
    • Does your throat swell?
    • Do you get abdominal pain?
    • What have you taken before that did or didn’t work?

    The best diagnostic test, he said, is still face-to-face history taking. Although even that can be pretty unreliable, he cautioned. Some people don’t know their family history; perhaps they’re adopted or estranged. There can also be what he called ‘variable penetrance,’ where some people have the mutation but have no symptoms. Or, in 25% of people with HAE Type I or Type II, it’s a ‘de novo’ mutation, which Prof. Riedl explained means, ‘You’re the first person in your family to have that mutation.’

    Without any abnormal tests, Prof. Riedl suggested that the first treatment for an unknown HAE or HAE-nC1INH is antihistamines, steroids, or preventative medicines such as omalizumab. These are, of course, treatments for allergies, but as Prof. Riedl put it, that’s the most common type of angioedema. Start there and move on to HAE medications if allergy treatment offers nothing. If people use an HAE medication and get better within a couple of hours after an attack, it can help clinicians understand the mechanism.

    Prof. Riedl concluded by acknowledging how troubling, disabling, and scary HAE is for people living with it. ‘We know there’s a lot of depression and anxiety associated with recurrent angioedema,’ he said.

    He left the audience with another Mark Twain quote: ‘Continuous improvement is better than delayed perfection,’ and a nod to the memory of Prof. Marcus Maurer in his commitment to continuing to fight the good fight.

  • Virtual Angioedema Centers

    ‘Within two minutes, Dr. Katelaris said: ‘I know what you’ve got, we just need to test for it.’ Fiona described her revelatory experience with Professor Connie Katelaris as she joined FIona onstage.

    Prof. Katelaris was asked to discuss her brainchild, Virtual Angioedema Centers (VAC). These were started in Prof. Katelaris’ home country of Australia, primarily as it is a vast country and sparsely populated, so there was a demand for knowledge about HAE from patients, relatives, and family doctors. The VACs mean people don’t have to travel for information.

    Prof. Katelaris stressed these are not medical consultations. They are a chance to offer impartial, independent, and authoritative information. Prof. Katelaris, or a nurse specialist, attends every virtual meeting. Questions have ranged from what happens with HAE in pregnancy to what I should ask my doctor.

    By using their hospital’s systems, privacy is always maintained, personal details are not kept, and calls and questions aren’t logged, allowing patients to freely speak about their symptoms and concerns.

    Since its start in Australia, there have been VACs in South Africa and India, which are large countries with diverse communities and languages. In India, doctors with a particular dialect can be identified for calls in different regions.

    Fiona closed the conversation by referencing the great feedback received about the VACs and their accessibility regarding format and the information provided.

  • HAEi’s Patient Driven Research

    The joint audience of healthcare professionals, patient advocates, and pharmaceutical industry representatives welcomed HAEi’s Chief Scientific Officer, Debs Corcoran, onto the stage. She and Tony Castaldo wanted to tell people about the power of patient-driven research.

    Tony set the scene by reminding everyone that while emotive patient stories have power, for decisions makers who decide on access to HAE medicines are motivated and convinced only by data and real-world evidence of disease burden and benefit of treatment.

    As a result, HAEi is at the forefront of producing data to support this decision-making, and Debs is in charge of all these projects.

    Debs told the audience: ‘Research could sound complicated, but in reality, it means that we bring together the data with your support. We do the analysis, and it’s all to give you our MOs the evidence to convince health ministries or insurers.’

    HAEi offers three core studies to MOs, these are:

    • The heat map survey
    • Baseline burden of illness study
    • Pharmacoeconomic, socioeconomic, and quality of life assessment

    Tony asked Debs to tell the audience all about the heat map survey. She told the audience that the heat map survey:

    • Is for countries where there are few people diagnosed with HAE
    • Helps to identify pockets of people with symptoms of chronic angioedema so that you can effectively target HAE education and awareness
    • Leads to more diagnosed patients and helps increase the priority health services places on HAE
    • Has already been done in the Philippines, with further studies planned in Thailand, Vietnam, Bangladesh and the Republic of the Congo

    Moving on to the baseline burden of illness study, this is for countries where there are patients with HAE but little or no access to modern HAE medicines. Debs took up the job of explaining in more detail. ‘This study turns patient experience into evidence of unmet need,’ she told the audience.

    The survey seeks to quantify:

    • Number of attacks
    • Emergency room visits
    • Hospitalizations
    • Patient quality of life

    Debs told them that the end product is evidence to convince your health service that people with HAE need better care. This study has been conducted in India, Mexico, and South Africa. Work is ongoing to bring this study to the Phillippines, Vietnam, Thailand, and China.

    As evidence of success, the Mexican data was used in a presentation to politicians, leading to significant benefits for HAE patients. Similarly, in South Africa, the data was accepted as an oral presentation at a rare disease conference (when HAE South Africa submitted it for only a poster!).

    Finally, the pharmacoeconomic, socioeconomic, and quality of life assessment analysis.

    Here, Debs highlighted two main aims. First, to quantify the overall economic value of modern HAE medicines, such as the ability to work, learn, and care for families. The second was to show the often dramatic quality of life improvements for people with access to modern HAE medication.

    This study was completed in the US, Nordic countries, Australia, New Zealand, and the United Kingdom. Projects are ongoing in Germany, Austria, and Switzerland.

    The goal is always to have the data published in a medical journal to maximize its visibility and credibility; it was an outstanding achievement that we published the US PE data in March 2021.

    >> Read the publication ‘Assessing the cost and quality-of-life impact of on-demand-only medications for adults with hereditary angioedema

    ‘We need to have a seat at the table; having publications is a surefire way to get that.’

    – Tony Castaldo, Chief Executive Officer, HAEi

    Debs and Tony said that the State of Management reports add a fourth dimension to our patient-driven research. This research asks leaders of MOs to provide a snapshot of:

    • Medication availability and reimbursement
    • Government health priorities
    • Challenges faced by people with HAE in their country

    HAEi published the reports on the HAEi website, and additional State of Management surveys are now in-process.

    Debs asked every advocacy leader to think about how they could get involved in research. Interested readers can contact Debs via d.corcoran@haei.org

    ‘We cannot conduct research without you, and we cannot conduct research without the people in your organization.’

    – Debs Corcoran, Chief Scientific Officer, HAEi 

  • Navigating the future of HAE: Ask the experts

    Probably the most eagerly anticipated Saturday session, Ask the Experts, saw Professor Henrietta Farkas, Professor Anete Grumach, Dr. Teresa Caballero, Professor Connie Katelaris, and Professor Marc Riedl join Professor Markus Magerl on stage. The questions came thick and fast, with Prof. Magerl trying to keep answers below 45 seconds.

    An audience member asked if her son, who has HAE but no symptoms, could be a pilot.
    The consensus from the group was that this would be possible if the young man were on modern, long-term prophylaxis to ensure he was symptom-free in what might be precarious situations.

    How does menopause impact on HAE?
    The experts responded that menopause causes lower levels of the hormone estrogen, which may be expected to lessen HAE symptoms, but this is not the case. Like pregnancy, there can be an improvement, worsening, or no change in symptoms. We cannot predict what will happen to attack frequency during menopause, so more research is needed.

    Does HAE make the person more susceptible to other diseases?
    The panel agreed there are no clear associations with other diseases.

    There has been some concern about a slightly increased risk of autoimmune diseases, but this was one old study. Additionally, there have been tests in animals that showed a slightly increased risk of blood clots, but this was not enough to prove it would happen in people with HAE.

    A study of people with HAE and COVID showed no increased susceptibility to COVID. However, for some people, worry and anxiety about getting other health conditions did seem to impact HAE symptoms negatively.

    Who takes care of the psychological aspects?
    While the experts believed that psychological support should be available to all people with HAE, there was an acknowledgment that a better job needs to be done. Often, too little is done, the panel felt, to address the mental health aspects of HAE. Having psychologists and genetic counselors available can be beneficial.

    Can I get a tattoo with HAE?
    The panel stated that if the patient was well controlled on modern prophylaxis, absolutely yes. If the tattoo were to be around the neck region, short-term prophylaxis should be considered.

    Is it possible to become addicted to HAE medication?
    In the bad old days, the panel suggested, patients were treated only with painkillers like opioids. These do nothing to treat the underlying swelling, and of course, opioids are addictive. Modern HAE-specific therapies cannot cause addiction.

    What’s the best treatment for a pregnant patient?
    As a specialist in HAE and pregnancy, Dr. Caballero responded. She felt that plasma-derived C1 inhibitor is the only treatment advised for a pregnant patient.

    If surgery is needed, do I need short-term prophylaxis even if controlled on lanadelumab?
    The panel noted that there is no data from the studies to date. Their thoughts were that patients who are very well controlled on lanadelumab should not need short-term prophylaxis. However, there was caution due to the lack of data, and some experts still argued for using short-term prophylaxis. Either way, everyone agreed having acute therapy in the operating suite was really important.

    What advice can you give people with HAE moving to another country?
    The panel’s suggested the things to think about were:

    • Insurance. Most will cover HAE, but there’s a lot of paperwork involved. Never just assume you’ll be covered.
    • Talk to your current specialist. Ask them to connect you with a specialist in the new country.
    • Download HAE Companion, HAEi’s app for foreign travel

    If HAE is okay on androgens, should a patient be moved to modern therapy?
    The panel accepted that there are still many countries with no access to modern therapy, although that is improving a lot.

    With androgens, even if it’s low dose and there are no apparent side effects, there could be hidden harms such as to blood pressure, cardiovascular problems, and liver function. The primary concerns are about long-term effects such as these. The panel recommended careful monitoring for anyone prescribed androgens.

    When will gene therapy be available worldwide? Will it mean the end of HAEi?
    The panel took the scientific question. In general, it will likely be some years before gene therapy is available outside of clinical trials, the first of which is due to be reported in 2026. In the panel’s opinion, this will be because regulatory authorities will need to see longer-term safety data due to the permanent nature of gene medicines.

    The next question will be about the cost of what are known to be costly therapies. How we pay for these treatments will be complicated.

    Responding on behalf of HAEi, Fiona Wardman was clear that while HAEi wouldn’t be around if HAE were no longer around, this wouldn’t be the case. There will continue to be new mutations. Some people will have HAE, and as a result, HAEi will be there every step of the way.

    This answer prompted the panel to make clear that gene therapies don’t eliminate the risk of passing the genetic mutation onto children. Even if the person treated with gene therapy no longer has HAE symptoms, their child could inherit the mutated gene. And, of course, we continue to have patients with spontaneous mutations.

    Will I have attacks if I go on the gene therapy trial?
    The available data shows that patients may have some attacks in the early stages. These quickly fall away, and the latest data shows an almost complete elimination of attacks.

    In the new study, there is a placebo arm, which means some patients will need to treat attacks with acute treatment. These individuals will have the option to have another option after six months and may receive the gene therapy.

    Why aren’t bradykinin levels measured to diagnose HAE?
    Bradykinin is important in multiple forms of HAE. Historically, it’s been tough to measure. It essentially disappears very quickly once blood is removed from the body. Some laboratories have published new ways to try and measure bradykinin, so it’s conceivable we may be able to measure bradykinin levels in the not-too-distant future, perhaps to diagnose or monitor how patients are doing on treatment.

    What is the role of kallikrein, and are there any effects if it’s blocked continuously?
    There were worries initially about blocking kallikrein, as the impact of doing this was still being determined. However, by the end of clinical trials, even long-term ones, there were no problems seen. Also, there is evidence that kallikrein is not entirely blocked; there is still some activity.

    Should every HAE patient have a genetic test at diagnosis?
    The panel felt that if the patient has HAE with C1 inhibitor deficiency, the diagnosis can be confident and confirmed without needing a genetic test. However, referencing Marc’s earlier talk, the panel suggested that with some mutations, it can be challenging to differentiate types of HAE, so a genetic test can provide certainty for patients. Additionally, genetic testing for children, new or unborn, can diagnose HAE where other tests may not work.

    Could a baby born with abdominal and genital edema have had an attack during a traumatic birth?
    The panel felt it was hard to be sure, but it is a likely explanation.

    What treatment is recommended for HAE with normal C1 inhibitor (HAE-nC1INH)?
    This is, as Marc Riedl’s presentation showed, a difficult question to answer due to a lack of data. It looks like icatibant and plasma-derived C1-inhibitor work for most patients to treat attacks. Tranexamic acid is often very helpful in preventing attacks, and depending on the mutation, there may be roles for some of the more modern treatments, such as lanadelumab.

    The final question to the panel was: How optimistic are you about the future of HAE?
    The entire panel was clear; the answer was: ‘Very!’

  • All about the youth

    Last but by no means least, Debs Corcoran returned to the stage with HAEi’s Co-Ordinator for the HAEi Youngsters’ Community (YC), Victoria Schultz Boysen. Their job, they revealed, was to say a little bit about the importance of the HAEi Youngsters’ Community and the youth advocacy program HAEi LEAP.

    Debs and Victoria started by acknowledging the fantastic work of Nevena Tsutsumanova, who is instrumental in the work of the HAEi YC. At this GLW, Nevena sat in the audience, not on stage, as she’s currently on maternity leave.

    Victoria highlighted key elements of the HAEi YC. There is an eight-member Youngster Advisory Group (YAG).

    >> You can find out more about the YAG here

    The composition of the advisory board changes over time. It is natural, especially as people grow older and ‘age out’ from the YAG.

    Victoria stated the aims of the HAEi YC were:

    • To be a place for young people around the world to find connection
    • To be a hub of information
    • To help youngsters find friends around the world
    • To empower people on their HAE journey
    • To share people’s uniqueness with the world
    • To welcome everyone and support each other
    • To show we are proud to be part of the global HAEi family
    • To share stories ideas and have the opportunity to travel
    • To never stop learning

    The latest projects- a social media internship and podcast called “HAE Let’s Talk”- were discussed. The HAEi LEAP 2025 program was launched to the audience. For 2025, HAEi is again seeking committed future advocacy leaders to be part of their education program. LEAP provides young people with skills for life and advocacy, and partners them with their local MO to undertake a project that can support their community.

    >> You can read more about these initiatives for our HAEi YC as part of the Youngsters at GLW write-up here

  • Behind the scenes heroes

    750 participants across two days. Dozens of lectures and sessions. Science. Advocacy. Speakers. Chairs (both the human type and the things you sit on). IT is on a grand scale, with simultaneous translation, giant presentation screens, and microphones of all types. Hotel accommodation, including food and drink, for a small army. It can be hard to see beyond the big stuff, but behind it all is a small and dedicated team making HAEi meetings like the GLW and GAF run smoothly. Global Perspectives asked a few behind-the-scenes heroes to step briefly into the limelight and tell you their experiences.

    Ole Frølich Christensen
    Ole is the Chief Technology Officer for HAEi. At the conference, you’re most likely to see him hunched over a laptop making sure Worldly is running smoothly, but he’s responsible for the many technologies and tools provided by HAEi, including HAE TrackR and HAEi Connect.

    Tell me what it’s like to work on an HAEi conference.
    Working together as a team where each has their specialty is great. We work together to solve the inevitable and unforeseen challenges in the best way possible.

    What were you looking forward to at this GLW/GAF?
    I really look forward to meeting the Member Organization leads to strengthen our collaboration with them even more. Part of this is giving people help with any of our online platforms, but they also give us invaluable feedback on improving what we have.

    What’s the best bit about HAEi conferences?
    Team spirit.

    What are the biggest challenges, and how are these overcome?
    There are always last-minute changes (in the program, running order, and arrivals/departures). We also have to work closely together to ensure everyone knows what’s happening.

    Karina Langsager
    Karina is HAEi’s Manager for Events and Conferences. As a result, much of the logistical responsibility for successful meetings like the GLW falls on her. Sadly, she couldn’t be at this conference due to ill health, but you can expect to see her colorful sneakers pounding the venue floors, ensuring the details are taken care of.

    Tell me what it’s like to work on an HAEi conference.
    It is a highly rewarding and dynamic experience. To be able to share information that helps people advocate for better in their country is incredibly rewarding. It literally improves the quality of life for people living with HAE all over the world.

    What were you looking forward to at this GLW/GAF?
    I was really looking forward to gathering with Member Organization leaders from all over the world. It is incredible to reconnect with so many and feel the strength of our community.

    What’s the best bit about HAEi conferences?
    I need to say the team. The conference team always supports each other, works tirelessly behind the scenes, and comes together with a shared purpose to ensure everything runs smoothly. Their dedication, collaboration, and positive energy make every event not just successful, but truly special.

    What are the biggest challenges, and how are these overcome?
    One of the biggest challenges is coordinating such a large, international event, especially with attendees from different time zones, languages, and backgrounds. Ensuring that everyone feels included and engaged all the way through, can be complex. We overcome this by planning meticulously, relying on strong teamwork, and leveraging technology to provide translation services. Another challenge is adapting to last-minute changes, whether technical issues or schedule adjustments. The key to overcoming this is our team’s flexibility and problem-solving skills, which ensure everything stays on track and that attendees have the best experience possible.

    Morten Thøgersen
    His job title at HAEi says IT specialist, and he’s the ‘Viking warrior’ of IT security mentioned by Michal. But you’re as likely to see this Scandinavian giant on the reception of the sign-in desk of an HAEi conference, as you are seeing him defending the IT servers from attack.

    Tell me what it’s like to work on an HAEi conference.
    Working on an HAEi conference is hard work, but seeing how we make a difference in patient and caregiver lives is exciting and rewarding.

    What were you looking forward to at this GLW/GAF?
    I am looking forward to our Member Organization leads from around the world joining up and sharing experiences.

    What’s the best bit about HAEi conferences?
    For me, the best bit is connecting with our participants, some of whom I have come to know over the years, and our amazing HAEi staff. Being a small part of a team that works so hard to help is inspiring.

    What are the biggest challenges, and how are these overcome?
    The biggest challenge is the logistics of assembling 750 people from around the world for three days. We have an amazing team that gets the job done over and over again.

    Rikke Sørensen
    Rikke is our Manager of Communications, Design and Graphics. Her work in the run-up to an event includes designing the entire program, hundreds of slides, and elements, like the signage that gets participants to the right place and the right time. On-site, she’s next to the audio-visual team, ensuring last-minute changes occur and that everything looks and feels perfect.

    Tell me what it’s like to work on an HAEi conference.
    Working on a big event like our global workshops and regional conferences is a team effort where we all want to send home people with new skills, new connections, and the power to make a difference in their own organizations or lives.

    What were you looking forward to at this GLW/GAF?
    We’ve put together an extensive, exciting, and diverse program that tries to listen to all the feedback we’ve received. To feel it gives participants what they want is what I look forward to the most.

    What’s the best bit about HAEi conferences?
    Hearing from Member Organizations, patients, and caregivers. And the party on Saturday night ;-)

    What are the biggest challenges, and how are these overcome?
    We have so much to tell and many important programs; the biggest challenge is to cover everything. The team that puts together the program works hard to ensure we meet peoples’ expectations around content and networking.

    Debs Corcoran
    As you may have seen elsewhere in the write-up of the HAEi Global Leadership Workshop, Debs is HAEi’s Chief Scientific Officer. She is in charge of planning and delivery of all the scientific elements of HAEi’s conferences. In Copenhagen, that meant taking on the first ACARE GAF, the largest-ever HAEi scientific meeting. Although she appears on stage occasionally, her ability to charm, convince, and cajole scientific experts makes her a ‘behind-the-scenes’ hero.

    Tell me what it’s like to work on an HAEi conference.
    It will be no surprise to hear that organizing an HAEi conference involves a lot of hard work from everyone! There’s creativity, excitement, challenges, problem-solving, laughter, a lot of Excel (possibly just me!), and a collective passion to make sure that each event is as useful as we can make it for the audience.

    What were you looking forward to at this GLW/GAF?
    My main role was organizing the GAF. It’s the first one and has similarities and differences to our conference’s scientific tracks. I was looking forward to seeing the rich variety of topics presented by our Faculty; our largest-ever poster session (to date), and how Angioedema Jeopardy and the Interactive Patient Journey would come to life!

    What’s the best bit about HAEi conferences?
    I’m not sure I can sum all that into one ‘best bit’, so how about:

    1. Our HAE experts who give us so much of their time and support
    2. Seeing old friends and making new ones
    3. The team that puts the meetings together
    4. The support we have from the industry to make the events happen
    5. Hearing how participating in the meeting positively impacts people’s lives, whether patients, caregivers, or healthcare professionals.

    What are the biggest challenges, and how are these overcome?
    In the last weeks of planning the GAF, we heard the unbelievably sad news that we’d lost Professor Marcus Maurer. The way the HAE patient and physician community came together to continue Marcus’ legacy and deliver a phenomenal first GAF shows that when we work together, we can achieve anything.

    Rachel Annals
    She’s an HAEi board member, but Rachel is the Swiss army knife of the HAEi on-site team. In addition to her role as Co-ordinator for Global Advocacy and ACARE, in Copenhagen she was embedded with Debs in the GAF, helping manage attendance from over 200 healthcare professionals.

    Tell me what it’s like to work on an HAEi conference.
    Working on an HAEi conference is exciting. It starts with being part of the operations team planning agendas, presenters, and logistics of the meeting, as well as making sure all the tech is in place to support our wide range of attendees from different countries and languages. It’s a very busy time, especially in the last few weeks before the event, but seeing the whole event come together and receive great feedback is fantastic. It makes all the time and hard work worthwhile.

    What were you looking forward to at this GLW/GAF?
    This year, I was lucky to be part of the GAF, which gave me a fantastic opportunity to meet and talk with lots of our HAE medical professionals from around the world and listen to their many presentations on HAE and other types of angioedema. It was an extremely busy couple of days, but so much information was shared and learned.

    What’s the best bit about HAEi conferences?
    The best part about the HAEi conferences is the opportunities for networking with other HAE Member Organizations and medical professionals worldwide, combined with the many presentations and discussions. There’s always something to learn and put into practice.

    What are the biggest challenges, and how are these overcome?
    The biggest challenge is to keep making the conferences bigger and better. With the growing number of Member Organizations and medical professionals learning about HAE, the event will undoubtedly grow bigger and more popular each time. Finding a venue to host our event may become more challenging, but it is equally impressive that more people know about HAE and that our advocacy work is paying off!

Thanks to our supporters

  • Update from the HAEi Youngsters’ Community

    HAEi Youngsters’ Community at GLW!

    The HAEi Youngsters’ Advisory Group (YAG) had the incredible opportunity to meet at the 2024 HAEi Global Leadership Workshop for a specific YAG-dedicated session to dive into current projects, brainstorm future ideas, and create tons of content for our socials. While Zoom has its perks, nothing beats the creativity and energy that sparks when we are face-to-face!

    The HAEi YAG currently consists of 8 members: Jacob (Canada), Dominika (Poland), Eirini (Greece), Isabel (United States), Kamila (Peru), Jess (Australia), Winona (South Africa), and Ania (Poland).

    Reuniting at GLW, YAG members interviewed youngsters from Member Organizations (MO) worldwide, snapped photos and videos, and set up a stand with our HAEi Youngsters’ Community postcards. Everyone could write messages to their younger selves or other youngsters, sum up the HAEi Youngsters’ Community in one word, or share dreams and hopes for the future.

    We are truly grateful for every postcard we received during the Workshop!

    On Saturday, Victoria Schultz-Boysen took to the stage to present some exciting projects within the HAEi Youngsters’ Community. The first, the HAEi Youngsters’ Community Social Media Internship Program, kicked off in January 2024, with the first round running from January to May. 4 amazing interns did a fantastic job creating engaging and exciting content for the Youngsters’ Community on Instagram.

    This is what some of the interns said:

    “It was a pleasure to be part of this program; I have learned a lot of things.”

    “I could share my ideas on social media and increase my knowledge about it.”

    The first round was such a success that a second round is now underway. This new round began in August and will continue through December. 5 incredible interns are taking charge of the Youngsters’ Instagram and working hard to keep the content engaging and creative.

    >> See the interns in action by following the Youngsters’ Instagram

    Another exciting project shared on stage was the HAE Let’s Talk podcast, which went live in mid-September. The podcast is one of the bigger projects within the HAEi Youngsters’ Community this year, and we are super excited to have it up and running! In the podcast, we chat about life, adventures, advocacy, and HAE. There’s a dedicated page on the Youngster’s website where you can read more and listen to the episodes.

    >> Tune in and listen to HAE Let’s Talk!

    On stage, Victoria Schultz-Boysen and Debs Corcoran shared some thrilling news: LEAP is back for 2025, and applications are open!

    HAEi LEAP is an educational program developed by HAEi that allows young people to learn new skills and develop as individuals and advocates.

    During the GLW presentation, Debs gave the audience a rundown of LEAP, sharing the key details. She also highlighted some fantastic projects from the LEAP Class of 2023, including resources like a caregiver’s guide, a youth advocacy guide, the first Youngsters’ Summer Camp for one country, and even a hike on hae day :-)! Plus, Victoria, one of the 2023 students, shared some of her own LEAP journey.

    Many individuals have already completed LEAP, and we look forward to another wonderful class in 2025.

    >> You can find out more about HAEi LEAP here

    Still haven’t joined the HAEi Youngsters’ Community?

    The HAEi Youngsters’ Community is free to join and is open to young people with HAE, family members, caregivers, and friends who are part of their country’s HAE MO and are between 12 and 25 years old.

    >> Head over to our website, youngsters.haei.org, and join us ☺

Have you wondered how to get your HAE under control? HAE TrackR can help you!

Developed by fellow HAE patients at HAEi, HAE TrackR is an easy-to-use electronic diary designed to record your HAE treatments, HAE attacks, and the impact HAE has on your life and the lives of your loved ones.

HAE TrackR allows you to download a comprehensive report of your treatments and attacks, which can be used as a tool for you and your physician to manage your HAE.

Features of the app:

  • Records your treatments (preventative, on-demand and clinical trial medication)
  • Records attacks and developments/improvements of attacks
  • Has a smart reminder functionality for prophylactic treatments
  • Is endorsed by ACARE for its ease of collecting and using data and comprehensive reporting
  • Is safe and secure, product and company neutral with no commercial interests

News from HAEi Countries Around the Globe

  • News from Afghanistan

    My name is Khatera Naimi, and I am the representative of Afghan HAE patients. I was supposed to participate in this conference, but unfortunately, due to barriers in visa issuance, I could not come to Denmark and join you all.

    I represent a vulnerable group of patients who have no supporters in Afghanistan. Additionally, I am a sick mother with four children, two of whom also suffer from HAE.

    I appreciate and congratulate the launching of this conference and hope this initiative continues.

    As a representative of my nation, I would like to share the concerns and requirements of my country’s HAE patients:

    1. In Afghanistan, HAE disease is still not completely recognized, a comprehensive diagnostic health clinic is not available, and most doctors still do not know about this disease.
    2. The de facto government also does not take care of this issue.
    3. There are no awareness sessions through the health ministry or media.
    4. Patients always suffer from wrong diagnoses and incorrect prescriptions and medications.
    5. Most patients are living below the poverty line and are not able to visit doctors.

  • News from Australia and New Zealand

    The last couple of months have been a vibrant period for HAE Australasia, marked by growth, education, and community engagement. Here are some of the key highlights from this time.

    Membership Growth

    HAE Australasia has seen encouraging growth in its membership, welcoming 10 new members over the past two months. This increase reflects the organisation’s commitment to providing support and resources for individuals and families affected by Hereditary Angioedema (HAE).

    Educational Webinars

    In July, HAE Australasia hosted the second webinar of the year, focused on women and children affected by HAE. The session featured Professor Connie Katelaris, who provided valuable insights on HAE in relation to women’s health and pregnancy. Additionally, paediatric immunologist Dr. Pinzon Charry was a special guest, sharing crucial information on children’s health with HAE, including testing and treatment guidelines.

    We have another engaging webinar scheduled for October 16th. This session will focus on patient stories and highlight helpful tools that all members should be using to better track, understand and manage their HAE. By sharing personal experiences and practical resources, we aim to empower our community and enhance overall management of the condition.

    The success of our webinars this year has been remarkable, prompting Zoe to create a dedicated webinar hub on the HAE Australasia website. Members can now easily access slides from past webinars, ensuring that valuable information is just a click away. Additionally, there’s a suggestion form available for members to propose future webinar topics, allowing us to tailor our offerings to the needs and interests of our community.

    Travel Grants for the 2025 HAEi Regional Conference APAC

    We have had an incredible response to our recent call for travel grants applications, aimed at patients and their caregivers to attend the 2025 HAEi Regional Conference APAC, in Manila, Philippines. We will soon commence the process of allocating grants by picking names out of a hat for as many members as possible.

    The upcoming conference promises to be an enriching experience, offering a valuable opportunity for our members to interact with peers from across the region. Attendees will have the chance to engage with experts, share experiences, and learn about the latest treatments and advancements in HAE management.

    2024 HAEi Global Leadership Workshop, Copenhagen

    Most of the HAE Australasia board has just returned from the 2024 HAEi Global Leadership Workshop (GLW) in Denmark. The GLW was held concurrently with the ACARE Global Angioedema Forum 2024 (GAF). With 750 participants from member organizations, physicians, and pharmaceutical companies representing 100 countries, the event provided a unique platform for collaboration and knowledge exchange.

    During the workshop, attendees learned about several promising new medication trials and research advancements in the field of HAE. Discussions highlighted significant discrepancies in prophylaxis and on-demand treatment options across different countries, emphasizing the ongoing challenges faced by patients worldwide. However, there was a sense of encouragement as various member organizations, HAEi, and pharmaceutical companies demonstrated a collective commitment to bridging these gaps in treatment access.

    The enthusiasm and dedication exhibited by participants to strive for equality in care, regardless of geographical location, reaffirmed the importance of global collaboration. HAE Australasia is excited to bring these insights back to our community and continue advocating for improved treatment options and support for all individuals affected by HAE.

    HAE Australasia Website Updates

    The HAE Australasia website has undergone several upgrades aimed at enhancing member resources and accessibility. A new “Youngsters” page has been added, providing engaging content tailored specifically for younger members. Additionally, downloadable Brady Club books are now available, offering fun and informative materials for children and families affected by HAE. This space is ever-evolving, and we are excited to see how this new page develops further.

    A brand-new Clinical Trials page has also been launched, keeping our members informed about ongoing trials they may be eligible to participate in. This resource is crucial for those interested in exploring new treatment options and contributing to important research in the field.

    It’s been an exciting and very busy couple of months for HAE Australasia, and we’re looking forward to the next couple of months as we wrap up 2024. With our upcoming webinar in October, a leadership strategy meeting in November, and continued growth within the organization, the future is shaping up to be even more promising.

  • News from Brazil

    Interview with Renata about Abranghe. During the 2024 HAEi Global Leadership Workshop in Copenhagen, Denmark, Abranghe had the opportunity to answer questions about the experience of its creation and the challenges faced during this period.

    We shared the motivation behind the creation of Abranghe, an organization dedicated to supporting patients and promoting awareness of HAE, with the voluntary involvement of patients and caregivers.

    To this end, we followed all legal procedures, gathering the necessary documentation and ensuring full compliance with Brazilian laws for formal registration as a non-profit organization.

    The creation of an assembly was necessary to approve the bylaws and, subsequently, obtain the CNPJ, a document that formalizes the organization’s legal existence, allowing its regular and recognized activities before the government and institutions.

    We also emphasize the importance of registering patients and medical resources to strengthen the association and achieve our goals.

    Regional group activity – South America and Mexico. The South America and Mexico group activity at the 2024 Global Leadership Workshop featured playful and creative dynamics. In a fun and interactive way, the leaders of the countries involved had the opportunity to test and develop their knowledge about AEH and share valuable information about the actions developed by the Associations of each country.

    The Quiz on AEH excitedly engaged everyone and allowed them to learn new things. In addition, the activity with the balloons encouraged the participants to reflect on their associations’ main challenges and seek solutions.

    It was an extremely productive afternoon for all participants.

    Raquel de Oliveira Martins, HAE Brazil, is co-author of the article Unmet needs in the management of hereditary angioedema from the perspective of Brazilian patients. The open-access version of the article – containing full bibliographic details – is now available online.

    >> You can read the article here

  • News from Canada

    HAE Canada’s summer started on a high note when we officially launched our new Strategic Plan and added it to our website. After months of dedicated brainstorming and strategizing, the HAE Canada Board of Directors and the HAE Canada staff successfully crafted our next five-year HAE Canada Strategic Plan. This comprehensive roadmap is a testament to our shared vision and commitment to advancing the mission of HAE Canada. Our new Strategic Plan meant updating our Vision and Mission:

    Our Vision
    That all Canadians with hereditary (HAE) and related angioedema achieve optimal health and well-being while living attack-free lives.

    Our Mission
    Provide support, education, and resources to our community, and advocate for exceptional disease management and equitable access to treatment.

    It’s a great achievement that reflects our collective passion and drive to make a lasting impact in the HAE community. A special thank you goes to Heather Dow, who expertly guided the board throughout the strategic planning sessions and enabled the team to come up with this fantastic plan.

    >> You can find our new Strat Plan on our website (in both English and French).

    In Canada, Canada’s Drug Agency (CDA), a not-for-profit health organization, provides advice to decision-makers (provincial and territorial governments) so they can make informed decisions regarding reimbursement for drugs in Canada. CDA’s advice is based on many different factors, including the patient’s perspective. Over the summer, HAEC provided CDA with two patient submissions: one to support garadacimab becoming available and one to support icatibant becoming available to Canadians with HAE with normal C1. Quebec has its own organization, Institut national d’excellence en santé et services sociaux (INESSS). We also provided a submission to INESSS for garadacimab. We want to extend a huge thank you to our members who shared their experiences with using these two treatments so we could create robust and informative submissions. We were thrilled with what we provided, and we are now anxiously waiting to hear if CDA decides to recommend reimbursement for these two effective treatments.

    In August, Michelle Cooper (HAEC President), Kerstyn Lane (Pacific Regional Director), Daphne Dumbrille (COO), and Heather Dow traveled to beautiful British Columbia to host a Patient Information Update in Vancouver. Joe Farago (Executive Director, Market Access) from Innovate Medicines provided an informative presentation explaining the path a drug follows, from when it’s first submitted to Health Canada until it reaches a patient. Next up was local allergist Dr. Peter Stapaniuk, who provided details about the specific process that a drug takes in BC, and he also discussed the latest HAE treatments that are “on the horizon”. Last but not least, Anthony Castaldo from HAE International presented about HAEi’s latest resources, specifically HAEi’s Advocacy Academy. We wish to extend a warm thank you to our presenters; they did an amazing job!

    Once the CDA gives its thumbs up and recommends that a new treatment should be reimbursed in Canada, the next step is for the manufacturer to negotiate a fair price with the Pan-Canadian Pharmaceutical Alliance (pCPA). In September, we were thrilled to learn that BioCryst Pharmaceuticals successfully completed negotiations with the pCPA for the oral, once-daily treatment ORLADEYO (berotralstat). This treatment is approved in Canada for the routine prevention of attacks of HAE in adults and pediatric patients 12 years of age and older. BioCryst can now work towards implementing public funding for ORLADEYO for eligible HAE patients. Canadian HAE patients are one step closer to accessing the first oral medication to treat HAE. There is still advocating work to be done, and HAE Canada is dedicated to helping Canadian patients access this new treatment.

    CDA’s Newborn Screening Advisory panel also requested feedback on the CDA discussion paper outlining its approach to developing newborn screening guidance. We had expert help from two of our member volunteers, Erin and Tracy, to prepare and submit a response in early September. We always greatly appreciate contributions from our volunteer members!

    Speaking of the CDA board members, Kim Speiss and Daphne were fortunate to attend the CDA-AMC’s 2024 Symposium in Ottawa in September. As always, CDA did a fantastic job hosting this important event, the first after introducing their new name & brand (formally known as CADTH). CDA discussed how they are working hard to incorporate patients’ experiences when making decisions on reimbursement for drugs and technologies in Canada. It was a wonderful opportunity to learn about CDA’s projects and initiatives while connecting with other patient groups, our sponsors, and Canadian physicians.

    The Canadian/International HAE Guidelines have been developed to ensure Canadians with HAE receive relevant and up-to-date care. Since the last Guidelines were released in 2019, new treatments and practices have been developed; therefore, leading Canadian and international HAE physicians gathered in Toronto in September to discuss how to update these Guidelines best.

    We want to extend a huge thank you to the Canadian HAE Network (CHAEN) for inviting HAE Canada to the meeting; Michelle, Kerstyn, and Daphne were fortunate to attend. We greatly appreciated being part of such an important and valuable process. We left knowing that the patient’s voice was truly listened to and heard; for that, we are grateful. Also, thank you to HAEC’s past president, Jacquie Badiou, who represented HAE Canada on the Guidelines Committee. She provided the patient’s perspective in the Guideline Committee meetings in the year leading up to the event.

    Throughout the weekend, we were continually reminded that HAE treaters are deeply dedicated to providing the best care possible for people with HAE. Seeing experts willing to volunteer their time for such an important cause was heartwarming. We are very much looking forward to seeing the final publication.

    Board members Michelle, Kerstyn, Kim, and Carmen Craciun (Treasurer), along with Daphne and Heather, were privileged to attend the 2024 HAEi Global Leadership Workshop in Copenhagen, Denmark, in October. Our fellow member organizations from across the globe, industry representatives, and leading HAE physicians were also attending the conference. The conference days were filled with learning, networking, and connecting with old and new friends. We were proud to contribute to the ACARE Global Angioedema Forum 2024, with Michelle presenting our poster titled HAE-nC1-INH: Treatment of Acute Attacks.

    We were thrilled that Michelle was invited to present at the session titled “Raising Awareness Through Media and Events.” She did a wonderful job summarizing our successful social media campaign to celebrate HAE Day 2024.

    Thank you, HAEi, for hosting this important and very worthwhile event. Bringing the community together in such a meaningful and productive way was truly appreciated. We left feeling energized and hopeful for a bright future for people with HAE.

    We are now gearing up for our next Patient Information Update on 19 October in Halifax, Nova Scotia. Our last Update in Halifax was in 2019, so we are excited to reconnect with our members on the East Coast!

  • News from Croatia

    Annual meeting of children suffering from HAE with doctors in 2024 – Tuheljske Toplice

    For the second year in a row, we organized a meeting of parents and children suffering from HAE with doctors.
    First of all, the most important ones, our children, were delighted, swimming pools, disco, magicians, trips, and socializing. We finally managed to gather many children of all ages from different parts of Croatia, as well as children from the neighboring countries of Slovenia, Serbia, BiH, and North Macedonia, at the pediatric week.

    As usual, we invited doctors to the meeting, who happily accepted the invitation. Direct contact between parents and pediatricians is also a great benefit of gatherings and the exchange of experiences between parents.

    All this happened in a wonderful spa, Tuheljske Spa.

  • News from Cuba

    2024 has been a year of progress for the community of patients with Hereditary Angioedema in Cuba.

    Our entry as a member country to the HAEi umbrella led to the participation of three patients and a doctor in the 2024 HAEi Regional Conference Americas, as well as one patient and two doctors in the 2024 HAEi Global Leadership Workshop and ACARE Global Angioedema Forum 2024 in Denmark.

    We have seen firsthand the dedication of HAEi to make us feel accompanied, and the regional advocates, in our case Javier Santana, are our voice before it.

    We are still one of the countries at a disadvantage due to the lack of diagnostic possibilities and specific treatments.

    However, the Cuban community is organizing itself – we already have a WhatsApp group made up of patients from various provinces of the country, through which we interact and share information, and we already have had the first virtual meeting.

    Recently, the possibility of diagnosing a group of people whose clinical symptoms suggest the presence of this rare disease has been opened.

    One step at a time, but without stopping, making ourselves visible is the watchword, and our work project is to walk the long road towards well-being and quality of life. We are sure that together we will achieve it!

  • News from Czech Republic

    Last call for artwork submissions for the HAE Junior exhibition. Art exhibitions featuring paintings by HAE juniors have been an effective, proven way to use creativity to raise HAE awareness among decision-makers, future doctors, and the general public. On hae day :-) 2024, the HAE Junior patient organization announced a call for artwork contributions for a new exhibition, which is open until 12th December 2024. This exhibition is planned to be on display as of 2025. The theme ‘My Inspiration’ seeks to display the heroes, real and fantastic characters, creatures, and joys that inspire young artists and support them in overcoming the difficulties caused by the rare disease HAE (and not only).

    We kindly invite children and adolescents (HAE patients, their siblings, and friends) from all over Europe up to the age of 18 to send us their artwork by 12th December 2024. We accept pictures done using watercolors, aquarelle, or tempera medium in A3 or A4 portrait/vertical layout.

    >> Please follow this link to find the submission form and learn more about the planned exhibition

  • News from Egypt

    Since the last issue of Global Perspectives, Takeda has officially registered TAKHZYRO in Egypt, and in the beginning of October, HAE Egypt attended the 2024 HAEi Global Leadership Workshop in Copenhagen, Denmark.

  • News from Hong Kong

    Dear HAE Community,

    As we reflect on the past year, we are thrilled to celebrate the 5th anniversary of HAE HK in August, marking a year filled with remarkable achievements and progress in our mission to support those affected by Hereditary Angioedema.

    Our journey over the past year has been nothing short of extraordinary, with significant milestones and events that have brought us closer together as a community. Here are some highlights from the past year:

    1. Full Reimbursement for On-Demand Medications: We are thrilled to announce that Hong Kong now offers full reimbursement for all on-demand medications for HAE, including C1-inhibitor replacement and icatibant. This means that patients can access these medications immediately without financial barriers, empowering them to manage HAE attacks effectively.
    2. Introduction of the Dry Blood Spot Test: At the patient and family day workshop on 8 June 2024 at the Sheraton Hong Kong, patients and their families had the opportunity to try the innovative Dry Blood Spot (DBS) test for HAE diagnosis. This new method promises greater convenience and accessibility in diagnosing HAE, marking a significant step forward in improving family screening and diagnosis.
    3. Community Engagement and Support: Our efforts to identify and support HAE patients in Hong Kong have been ongoing, with over 60 patients identified thus far. We continue to offer free testing and support to those who have not been diagnosed, emphasizing the importance of early detection and treatment.
    4. Oral drug trials: Exciting prospects for the development of oral emergency medicine have ignited hope and anticipation within the community.
    5. Introduction of HAE Patient Passport: We introduced the HAE passport to record attacks tailored for Hong Kong patients.

    Looking ahead, we are excited to be working on several new projects, with a particular focus on advocating for free preventative medication for patients in Hong Kong.

    We extend our heartfelt gratitude to our sponsors, partners, healthcare professionals, patients, and families for your unwavering dedication and support. Together, we stand united with love and compassion to empower patients and advance progress in enhancing the quality of life for those affected by HAE.

  • News from Hungary

    Dear fellow patients!

    HAE Hungary held its 2024 self-injection course on September 7, 2024, which was held by Dr. Imola Nagy and Judit Bali for 10 of our patients; thank you! During the course, the patients were able to practice on two artificial arms, which the Exceed Orphan company provided. We would also like to thank Zoltán Vilimszky and Tamás Vas for their cooperation and help. After practicing on the artificial arms, the patients could try their skills on themselves.

  • News from Mexico

    The last 2024 HAEi Global Leadership Workshop (GLW) and ACARE Global Angioedema Forum 2024 (GAF) held in Copenhagen have left us with greater knowledge and understanding of where and how we have to navigate now.

    The teachings and knowledge acquired in previous forums and workshops add to the news that was presented at this important GLW/GAF 2024 for the benefit of HAE patients, this will undoubtedly contribute to the dissemination of information about HAE among the medical community and among patients with suspicion of the disease who do not yet have an accurate diagnosis.

    The union between the members of the Mexican Association of Hereditary Angioedema (AMAEH) is very important to provide greater support to patients, caregivers, and relatives.

    An important aspect in which we are working as an association is to involve and invite the children of our community to the different activities we carry out, since they will be the ones who continue to navigate the ocean that we are building for them.

    AMAEH, as a member organization, the GLW/GAF 2024 encourages us to understand, train, and use among its leading members and other members the tools that we have at our disposal to have an orderly, precise, exact, and updated database.

    We are aware that the use of our social networks and website is of vital importance to promote knowledge and dissemination of the information we have about HAE, in such a way that we achieve accompaniment of the patient and caregiver at all times.

    Our main objective is aligned with the objective of the HAEi, which is to improve the quality of life of patients as well as have the medicine available in health institutions. Provide advice to patients, family members, and caregivers on how and where to obtain the medication.

    Continue to disseminate to the medical community what Hereditary Angioedema is and its treatment.

    All our love to heaven to Dr. Marco Cicardi and Dr. Marcus Maurer because without them, this would not have been possible.

  • News from Netherlands

    It’s hard to believe we’re already in the fourth quarter of the year! Time seems to have flown by. This past period has been filled with so many great moments. We’d love to share a few highlights with you.

    We would like to start by expressing our gratitude to HAEi for the incredible 2024 HAEi Global Leadership Workshop in Copenhagen. Three of the board members of HAE the Netherlands were participating. We enjoyed the excellent agenda full of inspiring sessions on a variety of subjects regarding managing a patient organization, making an impact and advocacy. The HAEi leadership continues to listen to our needs and provide accordingly. It was inspiring to listen to our fellow Member Organizations sharing their best practices on a variety of subjects concerning advocacy. We are proud that our president was invited to share her knowledge on building and managing an HAE patient organization.

    Between sessions, we enjoyed talking with old friends and making new ones. These conversations are always super inspiring. We went home with new energy and a lot of ideas to build a stronger organization, grow in numbers, and interact with our members who are not yet members.

    News from the Dutch medical community:

    In July, we received the exciting news that the Netherlands now has a second ACARE center: the UMCU in Utrecht. We’re very pleased with this development, and we see it as a step forward for the Dutch angioedema community. We’re looking forward to an even stronger collaboration with the angioedema team of the UMCU and would like to take this opportunity to congratulate them!  Exciting things are happening, and we’re very pleased with this progress!

    We’re also excited to announce that on Thursday, October 17th, Remy S. Petersen successfully defended her dissertation titled ‘Bradykinin-mediated Angioedema, The Path Towards Complete Disease Control’. Many of you might recall that Remy received the Young Investigators Award at the EMEA conference in Munich in 2023. We are incredibly proud of her achievements and immensely grateful for all she has done for us. We wish her the best of success in her future career and will miss her dearly in the HAE community.

    Now, on to the news from our organization.

    During the summer, we spent a lot of time brainstorming and planning activities to raise awareness and support for patients and their families. One project we worked on was a national survey. The last time a survey was rolled out was many years ago. A lot has changed since then. With our new survey, we hope to identify where information is lacking, for instance, about new treatment options. We believe it’s also a great way to find out how people in the Netherlands living with HAE experience living with this disease. With this kind of information, we will be able to improve our services. We’re hoping a lot of people will participate in this survey. If you’re living in the Netherlands and have HAE, or if you’re a parent of a child diagnosed with HAE, we invite you to take part in this survey. Click on the following link to go to the survey: Angio-oedeem enquete HAE/AAE – Google Formulieren

    And now, saving the best for last: On November 16, 2024, our annual Dutch patient day, titled “HAE and AAE: Moving Forward Together,” took place. Building on the success of last year’s event, we’re excited to make this year’s gathering even bigger and better. This time, we’re extending the invitation not just to Dutch patients but also to Dutch-speaking Belgian patients. We put together a compelling program with a blend of presentations and peer support sessions. The event was held at Hotel Van der Valk Breukelen from 10:00 to 16:00.

  • News from Panama

    The members of the board of directors of the Panamanian Association of Patients with Hereditary Angioedema, together with the medical advisor, Doctor Olga Melcina Barrera, were in a meeting on October 15 at the Central Office of the Ministry of Health with Dr. Jhonny Cuevas, coordinator of the Rare Diseases program in the country of Panama.

    In this meeting, some topics were discussed related to the progress that has been made in the Ministry of Health regarding the processes for the acquisition of medications for Hereditary Angioedema. For the beginning of the new year 2025, new meetings will be coordinated with Panamanian government officials in which specialist doctors on the subject of HAE, patients and representatives of HAEi will participate.

  • News from Paraguay

    In Paraguay, we are currently registering more than 20 patients already diagnosed in the country, free of charge and thanks to a study project in which several families were able to access the diagnosis without having to travel to another country, as many have done previously to have access to the diagnosis.

    We are also working from 2 areas: The public health system for the purchase of more appropriate and modern specific medications. This is possible through meetings with the Minister of Health in Paraguay, who referred us to a doctor in charge of strategies for rare diseases within the territory.

    In the private area, we are looking at the possibility of an agreement with the Ministry of Health to speed up the procedures and to be able to centralize the care of these patients since it has the necessary infrastructure to detect more patients with specific diagnoses, professionals with knowledge of pathology and interested in continuing training to be able to respond. Currently, some patients have already been admitted, one of them in a severe condition, with 30 days of intensive care for glottis edema.

    Paraguay’s participation in the 2024 HAEi Global Leadership Workshop, recently held in Copenhagen, Denmark, has been highly gratifying, as we bring new challenges and tools that we want to continue working on as a group of patients and caregivers. We would like to emphasize the gratitude of all of us to the international organization for this opportunity.

    With the commitment to continue moving forward, overcoming the difficulties of being a small country, but valuing each person whose life represents a family, a father, a mother of a family, a son/daughter, or a loved one within a home, we will not stop insisting on obtaining a medicine that in these times represents the quality of life for each one.

    Hoping to always contribute in the best way possible, I say goodbye and remain available for what we can contribute as representative members of our country.

  • News from Peru


    From August 16 to 18, we participated in the Rare Diseases Campaign in the city of Cajamarca, aimed at the general population. Dr. Oscar Calderón conducted screenings for Hereditary Angioedema. It was three days of raising awareness about HAE throughout the city.

    We supported Dr. Oscar Calderón with an informational booth at the XI International Congress of Immunology held on September 18, 19, and 20, where he participated as a speaker on the topic of Hereditary Angioedema.

    HAEi gave us the opportunity to meet in Copenhagen, Denmark, with leaders of patient associations with HAE from around the world. We thank HAEi for allowing us to participate in the Masterclass on “Effective Advocacy: Strategies for Local National Impact” at the 2024 Global Leadership Workshop (GLW) in Copenhagen, where we could share our experience in Peru with all the leaders. It was an intense and highly educational few days that enabled us to continue navigating towards the future.

  • News from Poland

    Hello HAE Friends,

    We are excited to share some key updates from our recent advocacy activities in Poland!

    At the end of Q2, HAE Poland (also known as the Swelling Beautifully Association) took part in several expert meetings, including discussions with representatives from the Ministry of Health, to advocate for the most pressing needs of Polish patients diagnosed with HAE. Key issues included ensuring access to long-term prophylaxis with subcutaneous C1-INH esterase for pregnant HAE patients and expanding the reimbursement criteria for preventive treatment with lanadelumab under the drug program. While both issues are highly complex, we are hopeful that, with the collective support of the entire local HAE community, we can secure reimbursement for these essential therapies. We kept working on the above in Q3; however, the goal hasn’t been achieved yet.

    At the start of the summer, the Swelling Beautifully Association hosted the 2024 Youngsters Summer Workshop, marking the second edition of this event. This initiative continues the original project from the HAEi LEAP Class of 2023, led by Dominika Słodka, a member of the HAEi Youngsters Advisory Group and a Regional Patient Advocate for HAE Poland.

    The 3-day workshop brought together 40 participants, including young patients, caregivers, and healthcare professionals. To enhance the program, Debs Corcoran, coach for the HAEi LEAP Educational Program, led the workshop, providing invaluable guidance. Debs empowered and motivated the young attendees and their families, addressing various aspects of patient advocacy in a well-structured and impactful way.

    During the workshop, Debs shared insights on time management – a vital skill that can help youngsters live more fulfilling lives while reducing stress, which can be a trigger for HAE attacks. She also led a session on advocacy, emphasizing that each of us is an advocate, and highlighted the importance of advocacy for those living with HAE. The youngsters actively participated, sharing their ideas and compiling a list of qualities that define the “ideal advocate.”

    Additionally, the participants collaborated to create guidelines for using social media to raise awareness about HAE. They discussed the role of social media in today’s world and brainstormed ideas for content to post on Instagram. Remember, by sharing our experiences and knowledge, we can create more meaningful content for social platforms.

    The summer workshop also provided an excellent opportunity to connect with expert physicians Dr. Marcin Stobiecki and Dr. Anna Dzieża-Grudnik. They helped the young patients deepen their understanding of HAE and provided a safe space to ask questions that may have previously felt uncomfortable or taboo. To build their advocacy skills, the youngsters even conducted interviews with the clinicians.

    The event concluded with an essential self-administration course, ensuring that every youngster with HAE, as well as their family members, feels confident in administering their treatment – whether intravenously or subcutaneously. This life-saving skill is vital for managing HAE independently.

    All the insights, knowledge, and guidelines gathered during the workshop will be used to prepare the second edition of the Polish Youngsters Magazine!

    Be sure to check out the following video from our 2024 HAE Poland Youngsters Summer Workshop: https://youtu.be/YUleYY-OzXc

    In addition to the summer workshop, from October 3-6, 2024, the HAE Poland Leadership team attended the 2024 Global Leadership Workshop in Copenhagen, Denmark. This incredible event brought together over 750 HAE stakeholders, including patient leaders, healthcare professionals, and industry representatives from 90 countries, with 16 attendees from Poland.

    Our Polish delegation took an active role in many sessions, making the most of this unique opportunity to network with other HAE advocates and expert physicians who participated in the ACARE Global Angioedema Forum 2024.

    Michał Rutkowski, Vice President of HAEi, Director of the HAEi Regional Patient Advocate Program, and President of HAE Poland, played an important role in the advocacy sessions, while Dominika Słodka was responsible for engaging young participants.

    HAE Poland regularly shares updates on our social media channels, keeping you informed about the Association’s latest activities and ongoing projects. We warmly invite you to follow us and stay connected!

  • News from Portugal

    Presence of Portugal at the 2024 HAEi Global Leadership Workshop

    Content-rich sessions brought speakers who, with stories of overcoming challenges and advancements in the field, touched and motivated everyone present. Each leader brought unique perspectives to the table, enriching the discussions and opening new paths for future collaborations and the development of our work.

    Our presence at this event was a privilege that filled our minds with ideas and our hearts with hope, significantly shaping the future of HAE treatment.

  • News from Russia

    Webinar

    On July 3 and 4, a webinar, “Life with HAE,” was held for patients and parents of children with HAE from Chelyabinsk and the Chelyabinsk region. Residents of other regions also joined the webinar, including new members of the organization who had recently joined the community. The meeting was moderated by the Chairperson of the Board of the International Public Organization “OPNAO” Elena Bezbozhnaya.

    Webinar

    July 22, 2024. The Interregional Public Organization “Society of Patients with Hereditary Angioedema” (IPO “OPNAO”) held an interactive webinar, during which OPNAO representatives answered patients’ questions. During the online meeting, members of the organization discussed the scheme and procedure for receiving subsidized drugs, talked about problems with drug provision in the regions, changes in legislation affecting patients with HAE, and much more.

    For some patients, the webinar was the first event of the IPO “OPNAO” in which they took part, so we tried to give patients as much useful basic information on drug provision and legal aspects as possible during the online meeting.

    Webinar

    We receive many questions from members of our community related to women’s health, pregnancy, and childbirth support for patients with NAO. Therefore, on August 27, we gathered everyone interested in the webinar “Women’s Health and Family Planning” and invited the best experts in this field to an online meeting. Doctors introduced participants to recommended treatment regimens and new diagnostic methods and answered the most pressing questions concerning patients.

    Social Survey

    We talk a lot and often with you about the risks associated with the lack of high-quality therapy and prevention; members of the community receive recommendations from experts to help provide themselves and their loved ones with the necessary medications. Understanding that the most vulnerable among us are children, we decided to find out what the current situation is with the provision of drugs to minor patients with HAE. To do this, we conducted an electronic survey among the parents of such children. Thanks to everyone who took part in our study. Thanks to you, we can assess the situation and see what changes we must work on together.

  • News from South Africa

    Our annual patient meeting was held on the 6th of April 2024 at the UCT Lung Institute in Cape Town and was attended by 67 patients, caregivers, and physicians. Topics covered included new medications, drug trials, pediatrics, genetics, and mental health. We had a successful, productive day learning more about HAE and meeting our new members. We received some excellent feedback from our attendees from our evaluation survey:

    “A very well-run and informative patient meeting; good to be made aware of new treatment options”

    “I have learned more about my HAE, looking forward to more engagement and growing awareness of HAE”

    “Very informative sessions and answered the most challenging questions that I had. It was a great experience, after having struggled with my child since birth who is now 16. Today’s engagement gave me insight and important awareness”

    Our mental health program “Mental Health Matters” was launched on HAE Day, and over the following six weeks, we posted a series of videos covering topics such as anxiety, depression, panic attacks, trauma, and self-care. The program, in partnership with Brittney Carmichael and Psynergy Counselling, addresses the emotional challenges faced by our patients, families, and caregivers. The video series can be viewed on the HAE S.A website as well as our social media channels.

    In October, our team attended the HAEi Global Leadership Conference in Copenhagen, where we were invited to share our perspectives on cultural sensitivity and stigma reduction during Saturday’s session. We had the opportunity to learn more about running a successful patient organization as well as meeting up with old and new colleagues and friends.

  • News from Switzerland

    We attended the 2024 Global Leadership Workshop in Copenhagen with 3 members of the Swiss HAE Association.

    We would like to thank the very good organization. The conference venue was also well chosen as it was right next to the airport. This meant that the journey to and from the airport was short as we had to leave very early on Sunday.

    CSL Behring expects approval in Switzerland for the active ingredient Garadacimab by the end of 2024. If approved, patients will benefit from an auto-injector (prefilled pen) for convenient administration.

  • News from Tunisia

    From Barhoun Arbia, HAE Tunisia

    The leader and some members of our organization attended the 2024 HAEi Global Leadership Workshop in Copenhagen in October. It was very important, useful, and full of information, and it was an opportunity to meet the leaders of other associations and exchange experiences.

    Soon, we will organize a meeting in our organization to prepare the logistics and planning of the Tunisian meeting with HAEi, which will be held in February 2025.

  • News from Türkiye

    What I learned in 10 years of HAE Advocacy

    I had the incredible opportunity to represent HAE Turkey at the 2024 HAEi Global Leadership Workshop (GLW), held from October 3-6, 2024, in Copenhagen, Denmark. The GLW brought together more than 750 participants from almost 100 countries.

    It was truly heartwarming to reconnect with friends worldwide, sharing our stories and experiences and nurturing these life-long friendships. That might sound strange, but having HAE and being part of this amazing, supportive global community makes me feel blessed.

    Our great moderator, Yong Hao, and dear friend Carla from HAE Peru

    I felt so proud and honored to be on stage to share our experiences. I was also honored to be a speaker in a masterclass, “Building Stronger Patient Communities: Working with Government, Case Studies, Strategies for Growth, Overcoming Barriers, and Effective Advocacy.”

    During my session, I shared some of the experiences and lessons we’ve gathered in our journey to raise awareness, improve treatment access, and foster collaboration for a rare disease called hereditary angioedema (HAE) in Turkey. The discussion of our session centered around four key questions:

    What inspired you and your organization to advocate for HAE nationally, and how did you know your efforts made a difference?

    We all started alone at the beginning; that was how I felt when I got my diagnosis. But I was lucky to find my community, my people. We united for the same goal: “improving the lives of people with HAE,” just as we all do in our respective countries.

    We were driven by a shared feeling of isolation and the need to change the misdiagnosis landscape.

    • National Awareness: Inspired by the sense of isolation, we raised national awareness about HAE, helping others avoid years of misdiagnosis and the burden of untreated HAE.
    • Improved Access to Treatments: Registered three new original medications—Ruconest, Berinert, and Takhzyro—along with four generic alternatives of icatibant, all domestically produced, ensuring a stable supply and more treatment options for the patients in Turkey. C1-Inhibitors are also available and reimbursed for acute attack and prophylaxis in some cases.
    • Participation in Clinical Trials: Over 20 participants, including some of the board members, joined clinical trials for modern medications, which led to improved quality of life for those with severe HAE attacks.
    • Strong Collaboration with Physicians and Authorities: Established strong partnerships with physicians and key authorities, including the Ministry of Health, the National Social Security Institution, The Turkish National Society of Allergy and Clinical Immunology, and the Turkish National Rare Diseases Association, amplifying the patient voice in Turkey’s HAE projects.
    • Guidance for Diagnosis and Treatment: Connected many individuals with specialists for diagnosis and treatment, ensuring timely and appropriate care for HAE patients. Delay in diagnosis is an issue and must always be observed with the physician network to improve it.

    How did you ensure your message about HAE reached people across the country?

    Turkey is a vast country, and within our context, collaborating with expert physicians nationwide has helped many patients receive accurate diagnoses and appropriate treatments. We are grateful and fortunate to maintain such excellent communication and cooperation with our medical professionals.

    • Diagnosis and Family Screening Campaign: Launched the “Diagnosis and Family Screening Campaign,” which helped over 300 individuals get tested, leading to more than 70 new HAE diagnoses.
    • Media Outreach: Utilized social media, internet platforms, and traditional media like magazines and newspapers to spread awareness across the country.
    • Public Awareness via Media: Collaborated with Alican Sabunsoy, a well-known TV figure, who shared his experience as an HAE patient on the national TV show “MasterChef,” resulting in widespread awareness and more than ten new diagnoses. An Instagram post about this reached over 57,000 people in Turkey. The YouTube video has been watched more than 300,000 times https://youtu.be/8hKqIy5-nzc?si=TnjOWquDilZl7CW7&t=309
    • Collaboration with National Associations: Partnered with the Turkish National Association of Allergy & Clinical Immunology (Türkiye Ulusal Alerji ve Klinik İmmünoloji Derneği) and the National Rare Diseases Association to train over 150 healthcare professionals, improving early diagnosis and proper treatment.
    • Policy Recommendations: Developed comprehensive policy recommendations on diagnosis delays, treatment access, work and education challenges, and social rights, and presented these to the Ministry of Health and other relevant authorities with the help of Sağlıklı Hayatı Teşvik ve Sağlık Politikaları Derneği.

    What challenges did you face, and how did you handle them?

    • Embracing the Baby Steps: We learned that significant change doesn’t happen overnight. It was important to celebrate small victories along the way—whether it was securing a single new diagnosis, establishing contact with a physician, or organizing our first patient meeting. These small wins kept us motivated and built momentum for larger accomplishments. By focusing on achievable milestones, we expanded our reach and impact gradually.
    • Breaking Down Barriers: We worked to change people’s perceptions of the association and HAE as a whole.
    • Most Attended Event in Association History: Organized an online event during the COVID-19 pandemic, attracting over 200 live participants. The number of attendees was even higher since families often joined together.
    • Supportive Online Environment: Created a virtual space for open dialogue where patients and caregivers could share stories, ask questions, and receive support during uncertain times. Extended the planned 1.5-hour session to nearly 4 hours based on participant engagement, ensuring everyone had a chance to ask questions and share experiences.

    What’s one key lesson you’ve learned, and how would you apply it to future efforts or advise others aiming for national impact?

    The broader your reach, the greater the efforts you’ll need to make.

    • Sustainable Systems: Establish sustainable and manageable systems for your initiatives. The broader your reach, the greater the efforts you’ll need to make. This has been a key learning from my 10 years of HAE advocacy experience and is crucial for all patient organizations in their advocacy journey.
    • Defined Processes and Action Planning: Establish a clear mission and set specific, realistic goals. Define the who, what, when, where, and how for processes and actions (e.g., posting at least one monthly informative post on social media).

    • Data-Driven Advocacy: Collected data on HAE prevalence, diagnosis delays, and treatment accessibility, which has been invaluable in policy discussions with the Ministry of Health. Strong data is the easiest way to support your arguments.
    • Team Training and Capacity Building: Invested in training programs to enhance skills in advocacy, legal matters, and project management for the team, ensuring continued growth.
    • Collaborations with Other NGOs and Universities: Established partnerships with universities to engage young advocates and researchers, expanding our organization’s reach and impact.

    I want to thank HAEi and all friends around the world for our united efforts to provide a better life for people with HAE.

    I’d love to hear your thoughts or connect with others working in patient advocacy. I’m learning every day, and I’m ready to learn from your experiences, too!

  • News from UK

    Our 2024 Patient Day in York was a great success; we had patients, healthcare professionals, and speakers all come together to share valuable information and experiences. Patients had the opportunity to learn more about their condition, treatments, and how to manage their health better; healthcare professionals could provide expert advice and support to patients, while our PhD student shared her research project with us all.

    There was time for meeting and talking to others, which attendees always say is one of the best parts of the day. Many enjoyed catching up with old friends, while others took the opportunity to strike up new conversations with those they hadn’t met before.

    During our lunch break, we walked along the river while wearing our HAE UK-branded t-shirts. The sun was shining, and the weather was perfect for a stroll. This walk was also an important opportunity to raise awareness about HAE. As we walked, we talked about our experiences and the challenges faced by people with HAE.

    Overall, the event was a fantastic opportunity for everyone involved to come together, learn from each other, and share experiences.

    2024 HAEi Global Leadership Workshop and ACARE Global Angioedema Forum 2024

    Six representatives from HAE UK, along with some UK doctors, recently participated in the 2024 Global Leadership Workshop for patient organization leaders and the ACARE Global Angioedema Forum 2024 for angioedema experts in Copenhagen, Denmark. During the workshop and forum, attendees could share their knowledge and experiences and learn from experts in the field. The event provided a valuable platform for networking and interaction with other HAEi member organizations, ultimately helping to further support and advocate for individuals affected by HAE. We are excited to start implementing some of the things we learned to support our UK patients and their families further.

    Clinical trials

    Several UK patients with HAE are currently participating in various clinical trials, and the results are incredibly promising. These trials are showing significant improvements in the management and treatment of HAE, giving hope to patients who have long struggled with this rare and potentially life-threatening condition.

  • News from Ukraine

    I am happy to share the autumn successes and events in the Ukrainian Association of Patients. In September, with Takeda, a series of interviews with patients and medical professionals was completed and published. On the website careforyou.com.ua, in the video section, short educational videos have already been posted for viewing on the following topics:

    • clinical manifestations of HAE,
    • HAE examinations,
    • a patient with HAE and his family,
    • in search of the causes of HAE,
    • interaction with medical professionals, treatment of an acute attack of HAE,
    • issues with access to HAE treatment and prevention of HAE attacks.

    Five patients and five doctors participated in the filming, and the result was amazing. This will help new patients and their families better understand hereditary angioedema, which is also an excellent guide for doctors who have not encountered this disease before.

    And, of course, I hasten to share our participation in the 2024 HAEi Global Leadership Workshop in Copenhagen, Denmark, October 3-6. Ukraine was represented at the conference by seven participants: two doctors and five patient community representatives.

    Based on an online survey of 43 patients, a report and poster for the scientific track were created with the help of Anastasia Bondarenko and Lyudmila Zabrodska. On behalf of the Ukrainian delegation, they presented updated data on the diagnostics and treatment of HAE in Ukraine and discussed challenges faced by medical professionals during the war.

    The average delay in establishing a diagnosis is 8-10 years worldwide and 10-15 years in Ukraine. However, it is crucial to know that efficient treatment exists and can significantly improve the patient’s condition if the diagnosis is made in a timely manner.

    The conference featured lively discussions on problematic issues of HAE differential diagnostics, comorbidities, and new promising solutions in treatment. In particular, one introduced the results of clinical trials of new oral medications (primarily injectable treatments are currently used) and gene therapy that could radically change the approach to treatment.

    This event is an important step towards improving the life quality of patients with HAE and raising awareness about this disease. We can achieve significant changes together.

  • News from USA

    HAEi Global Perspectives articles – Fall 2024

    Honoring HAEA Scholars: Highlights from the 2024 HAEA Scholarship Gala

    The 2024 HAEA Scholarship Gala was an evening to remember, bringing together outstanding scholarship recipients to celebrate their remarkable achievements. Held at the Hyatt Regency Washington on Capitol Hill in Washington, D.C. in July, the Gala celebrated academic achievement and community spirit while reinforcing the HAEA’s commitment to empowering and supporting the next generation of leaders.

    The Gala highlighted heartfelt speeches delivered by scholarship recipients who shared personal journeys, challenges, and aspirations for the future. Their stories are a testament to the resilience and determination of the HAEA community.

    You can watch the full Gala event at https://vimeo.com/992191547?.

    HAEA Advocates Voice Their Concerns During Capitol Hill Day Visits

    On July 15th, over 200 HAEA advocates visited 113 congressional offices on Capitol Hill to deliver a vitally important message requesting action on matters that affect the future of people with HAE and their families. During these meetings, our HAEA volunteer advocates voiced their support for legislation related to medication access and research funding to support new and improved therapies.

    HAEA advocates prepared for Capitol Hill Day by participating in an engaging training session where they learned about how to use their stories to influence policymakers.

    Thank you to all who attended the HAEA 2024 Capitol Hill Day! Your efforts to inform our elected representatives were well received and promise to have a positive impact on legislation that affects the HAEA community.

    You can watch a recap video of this event at https://vimeo.com/1006313150

    Breakthrough HAEA-Initiated Research on Quality of Life Published in a Prestigious Medical Journal

    An HAEA-initiated research project that included well over 500 HAEA friends has resulted in a ground-breaking HAE-specific tool that measures Quality of Life for people with HAE in the United States. The prestigious medical journal–Annals of Allergy, Asthma, and Immunology–recognized the value of this unique research by recently publishing the article we prepared that documents the development and testing of this groundbreaking tool. The study can be viewed by clicking HERE.

    Our Quality of Life tool is designed to benefit our community by  helping physicians and researchers:

    • Accurately captures the way HAE affects the everyday life of individuals and families,
    • Demonstrate (to health insurers and others) the value of life-changing improvements in health and quality of life that results from modern HAE medicines, and
    • Avoids the under-reporting of challenges faced by people with HAE in the US that is seen in other currently available Quality of Life tools.

    Please stay tuned for information on other exciting ongoing HAEA research projects that are all designed to help improve the health and well-being of everyone in our community.

    Celebrating Youth Advocacy Month by Recognizing Our Next Generation of Leaders

    Every October, we celebrate Youth Advocacy Month by showcasing the stories and activities of the youngest advocates in our community. This year, we are thrilled to announce the launch of the Youth Advocacy Achievement League, an elite group of young advocates who have stepped up to raise HAE awareness, advocated for legislative change, or made an impact in their local community. Over 30 remarkable young individuals have been nominated to be members of the League.

    You can learn more about their inspiring activities on the HAEA Youth instagram, @HAEAyouth, and celebrate the difference they are making as leaders and changemakers. These advocates are the next generation of HAEA leaders and their efforts serve as a powerful reminder of the importance of youth voices in driving meaningful change. Stay tuned to learn more about these amazing young people and join us in honoring their achievements throughout Youth Advocacy Month!

Clinical Trials Update

HAE-related scientific publications

  • In addition to a wide range of case reports and small series, here are summaries of recently published HAE-related scientific papers. Data search undertaken 3 October 2024.

    Current and emerging therapeutics in hereditary angioedema

    By Toan Do and Marc Riedl

    The authors review the current and future landscape of treatment for HAE. In doing so, they make clear that the goal of HAE therapy is to normalize the patient’s life and ultimately achieve total control of the condition; shared-decision making is essential to reaching this goal. They tell the reader that effective on-demand HAE medication should be used as early as possible during an angioedema attack for optimal efficacy in treating HAE symptoms; delays in on-demand treatment are a common cause of HAE morbidity. Finally, they state that long-term prophylactic (LTP) medications are increasingly used to manage HAE due to improvements in efficacy.

    (Immunology and Allergy Clinics of North America, August 2024)

    Factor XII structure-function relationships

    By Aleksandr Shamanaev, et al

    The authors investigated how the structure of Factor XII impacted on its function in the body. The authors conclude that FXII circulates in blood in a “closed” form that is resistant to activation until interactions with other substances produced by the body. The authors believe that this will have implications for understanding how FXII contributes to diseases such as hereditary angioedema and for developing treatments.

    (Seminars in Thrombosis and Hemostasis, October 2024)

    Epidemiology, economic, and humanistic burden of hereditary angioedema: A systematic review

    By Xin Guan, et al

    The authors of this paper look to calculate the burden of HAE on patients’ lives. By reviewing available literature on 10,310 patients with HAE, they indicated substantial financial costs, mostly due to missing work and loss of productivity. The authors conclude that early diagnosis and improved awareness amongst healthcare professionals can reduce the burden of HAE on patients and society.

    (Orphanet Journal of Rare Diseases, July 2024)

    Systemic inflammation biomarkers during angioedema attacks in hereditary angioedema

    By Johana Gil-Serrano, et al

    The authors looked for biomarkers of inflammation in people undergoing an HAE attack. They found that inflammation may extend beyond the area of swelling, and that the involvement of inflammation pathways in HAE needs further investigation.

    (Frontiers in Immunology, June 2024)

    Long term outcome of C1-esterase inhibitor deficiency

    By Luong Hoang Long, et al

    The authors review the evidence on long-term outcomes for people living with HAE. They conclude that in any annual health survey, people with HAE should be assessed for autoimmune diseases, cancer, and other chronic illnesses. In patients being treated with androgens, tests should also include blood fats and liver examinations.

    (Asian Pacific Journal of Allergy and Immunology, June 2024)

    Hereditary angioedema prevalence and satisfaction with prophylaxis in South Australia

    By Alexander Troelnikov, et al

    The authors report a lack of population studies amongst Australian HAE patients. Undertaking their own research, the authors conclude that the prevalence of HAE in south Australia is in line with international reports. They also demonstrate that patients on long-term prophylaxis report these to be effective and are highly satisfied.

    (The World Allergy Organization Journal, June 2024)

    Classification, diagnosis and pathology of angioedema without hives

    By Bruce Zuraw and Sandra Christiansen

    The authors conclude here that being able to efficiently categorize and diagnose all forms of angioedema results in improved patient outcomes.

    (Immunology and Allergy Clinics of North America, August 2024)

    Prophylaxis in hereditary angioedema: a United Kingdom Delphi consensus

    By Patrick FK Yong, et al

    The authors, which include HAEi’s very own Rachel Annals, looked to update the current consensus amongst UK clinicians and patient advocates on how to prevent attacks in people with HAE in the UK. The key points from the resulting consensus statement were:

    • UK access criteria for modern long-term prophylaxis based on numerical frequency of attacks alone are too simplistic and potentially disadvantage a cohort of patients who may benefit.
    • Patients should be seen in expert centers, remote monitoring of patients is popular post-pandemic, and the use of patient-reported outcome measures can potentially improve patient care.
    • Psychological health is an area in which patients may benefit, and recognition of this is important for future research and development.

    Hereditary angioedema: The clinical picture of excessive contact activation

    By Remy S Petersen, et al

    Based on a review of existing data, the authors conclude that while C1-inhibitor is an important regulator in several intravascular pathways, symptoms in HAE patients are caused by insufficient inhibition of the contact activation pathway and kallikrein-kinin system. The subsequent unpredictable and potentially life-threatening angioedema attacks have a pronounced effect on quality of life. The authors note that new, highly specific therapies have the potential to enable patients to lead a normal life.

    (Seminars in Thrombosis and Hemostatis, October 2024)

    Hereditary angioedema: Beyond swelling

    By Paul A Kyrle and Sabine Eichinger

    Writing in the journal Blood, the authors provide an editorial linked to the article below. They discuss the evidence linking HAE with blood clots in the veins of the legs (VTE or venous thromboembolism). If this is the case, they argue, then patients should be considered for prophylactic C1INH treatment after an unprovoked VTE. All patients with HAE who suffer a provoked VTE should be treated the same as any other patient.

    (Blood, July 2024)

    Increased risk of venous thromboembolism in young and middle-age individuals with hereditary angioedema: A family study

    By Linda Sundler Björkman, et al

    The authors investigated families with HAE in Sweden, to see if there was any increased incidence of blood clots in the veins of these people. They found that these blood clots were associated with young and middle-aged individuals with HAE. They conclude by suggesting that HAE may be considered a new, rare, abnormal tendency towards blood clot formation.

    (Blood, July 2024)

    Deucrictibant for angioedema due to acquired C1-inhibitor deficiency: A randomized-controlled trial

    By Remy S Petersen, et al

    The authors conducted a small trial to see how safe and effective a potential new treatment for HAE, called deucrictibant, could be at treating and preventing HAE attacks in patients with acquired C1-inhibitor deficiency. The authors conclude that deucrictibant has the potential to effectively and safely treat and prevent angioedema attacks due to C1-inhibitor deficiency.

    (Journal of Allergy and Clinical Immunology, July 2024)

    Physician and patient-reported outcomes by hereditary angioedema type: Data from a real-world study

    By John Anderson, et al

    The authors asked patients and clinicians about the severity of their disease and the outcomes from treatment. They conclude that patients with HAE with normal C1 have increased disease activity and severity compared to those with HAE type I and type II, leading to greater impairment to their quality of life.

    (Allergy and Asthma Proceedings, July 2024)

    Contact system activation and bradykinin generation in angioedema: Laboratory assessment and biomarker utilization

    By Sandra C Christiansen and Bruce L Zuraw

    Proteins in the blood can, when released, lead to blood clotting and inflammation. When these proteins are produced incorrectly or not at all, this can cause diseases such as HAE. In this review, the authors explain the evidence that bradykinin generation underlies even HAE with normal C1INH.

    (Immunology and Allergy Clinics of North America, August 2024)

    An LNP-CRISPR gene editing drug demonstrates efficacy and safety in patients with hereditary angioedema following in-vivo administration

    By Norbert Pardi and Zoltán Ivics

    In an editorial reviewing the recent data published by a team led by Hilary Longhurst, the authors discuss the potential for gene-editing drugs to prevent HAE attacks in people living with the disease. They conclude that such drugs may offer the extraordinary perspective of treating severe genetic conditions with a single-dose, one-and-done treatment.

    (The Journal of Allergy and Clinical Immunology, August 2024)

    Inter-α-trypsin inhibitor heavy chain 4 (ITIH4) as a compensatory protease inhibitor in hereditary angioedema

    By Anne Troldborg, et al

    The authors state that nothing is known about the role of inter-α-trypsin inhibitor heavy chain 4 (ITIH4) in HAE despite its involvement in many pathways that lead to attacks. The authors conclude that ITIH4 may be valuable as a diagnostic and prognostic biomarker in HAE.

    (The Journal of Allergy and Clinical Immunology, August 2024)

    Targeting factor XIIa for therapeutic interference with hereditary angioedema

    By Danny M Cohn and Thomas Renné

    The authors state that targeting factor XIIa is emerging as an attractive option for disrupting HAE attacks. They conclude, following a review of the treatments currently being evaluated in clinical trials, that there may be potential advantages to targeting factor XIIa versus other targets in the kallikrein-kinin system.

    (Journal of Internal Medicine, October 2024)

    Centralized care model for hereditary angioedema overcomes geographical barriers

    By Ashley Holmes, et al

    The authors aimed to see if there were differences in care between HAE patients living in urban or rural settings. They conclude that in their practice (an ACARE center), rural patients received similar high-quality care, which they attribute to the centralized care model employed in which HAE patients in the region are seen at a single comprehensive care clinic.

    (Frontiers of Immunology, July 2024)

    Structural basis for the inhibition of βFXIIa by garadacimab

    By Ieva Drulyte, et al

    The authors examined the structure of the potential HAE treatment garadacimab, using an electron microscope. They found garadacimab strikingly similar to true factor XIIa inhibitors such as C1 inhibitor.

    (Structure, July 2024)

    Hereditary angioedema: 24 years of experience in a Portuguese reference center

    By Cláudia Varandas, et al

    With no recently published studies in Portuguese patients, the authors examined records of people with HAE treated at a single center in Portugal. They concluded that distinguishing between HAE and other common causes of angioedema is vital, leading to reductions in diagnostic delay and improving management, outcomes, and quality of life.

    (European Annals of Allergy and Clinical Immunology, September 2024)

    Prodromal symptoms of hereditary angioedema (HAE) attacks: A patient survey in UK and Spain

    By Patrick FK Yong and Mar Guilarte

    The authors indicate that data shows that the majority of HAE patients experience prodromes, where there are signs of an impending attack. However, they feel that research into HAE prodromes is lacking, especially in terms of diagnosis and treatment. Following their research, the authors conclude that patient behavior around early treatment was highly variable, and that better guidance may address this. Additionally, they feel that better understanding of prodromes may help patients better prevent or act earlier to mitigate attacks.

    (Allergy, August 2024)

    Development and validation of a US quality of life instrument for hereditary angioedema due to C1 inhibitor deficiency

    By Anthony J Castaldo, et al

    The authors, including Anthony Castaldo and Deborah Corcoran from HAEi and HAEA, developed an HAE-specific quality-of-life instrument that takes account of the unique issues in the US healthcare system. The authors conclude that the new tool, called HAE-C1INH-QoL, is the first HAE-specific quality-of-life tool to be validated in the United States, making it more relevant to US patients with HAE than existing angioedema tools.

    (Annals of Allergy, Asthma and Immunology, July 2024)

    The CC2D2B is a novel genetic modifier of the clinical phenotype in patients with hereditary angioedema due to C1 inhibitor deficiency

    By Nina Rupar, et al

    In this genetic-based study, the authors looked for genes related to HAE in families from south eastern Europe. They conclude that their study demonstrated novel genetic factors leading to the variability of C1-INH-HAE. Additionally, they indicate the importance of genes called CC2D2B, CCNJ and ZNF518A.

    (Gene, August 2024)

    Antisense and CRISPR-based drugs build cases for better hereditary angioedema treatments

    By Asher Mullard

    With the potential new HAE treatments donidalorsen and NTLA-2002 on the horizon, the authors discuss whether a functional cure could be possible for HAE.

    (Nature Reviews Drug Discovery, July 2024)

    A human-centered innovative approach based on persona in hereditary angioedema

    By Francesca Perego, et al

    In this study, the authors investigated whether the process of human-centered design (HCD) could have a role in the care people with HAE receive. They conclude that using an innovative HCD approach based on the person being treated, will extend beyond their physical illness to address psychological and social aspects of the individual’s wellbeing.

    (Orphanet Journal of Rare Diseases, August 2024)

    Advent of oral medications for the treatment for hereditary angioedema

    By Anna Valerieva, et al

    The authors look into the possibility of oral treatments for HAE. They conclude that substantial advances have been made in developing oral treatments. They believe that these treatments have the potential to improve and optimize clinical outcomes, satisfaction, and quality of life among patients with HAE.

    (Clinical and Translational Allergy, September 2024)

    Altered levels of phospholipases C, diacylglycerols, endocannabinoids, and N-acylethanolamines in patients with hereditary angioedema due to FXII mutation

    By Anne Lise Ferrara, et al

    These authors investigated HAE due to mutations in the FXII gene. They conclude that phospholipase C may have a role as a potential biomarker or therapeutic target.

    (Allergy, June 2024)

    Validation and correlations of the Angioedema Activity Score (AAS), Angioedema Quality of Life (AE-QoL) questionnaire, and Angioedema Control Test (AECT) in Chinese patients with angioedema

    By Hugo WF Mak, et al

    The authors aimed to validate HAE-relevant quality-of-life tools in China. They conclude that their study showed that a series of tools are valid and reliable tools for use with Chinese patients.

    (Journal of Allergy and Clinical Immunology: Global, July 2024)

    Disparities in geography and race in hereditary angioedema: Analysis of 64 trials and 4354 patients

    By Hugo WF Mak, et al

    The authors indicate a significant disparity in the participation of non-Western and non-White participants in clinical trials for HAE. They believe further global efforts are necessary to improve inclusiveness and deliver more equitable HAE care.

    (The Journal of Allergy and Clinical Immunology: In Practice, July 2024)

    Early diagnosis of hereditary angioedema in Japan based on a US medical dataset: Algorithm development and validation

    By Kouhei Yamashita, et al

    The authors introduce their study by showing that, based on prevalence estimates, only 20% of potential HAE cases in Japan have been identified. The authors conclude that using an artificial intelligence model can detect HAE in patients with typical symptoms.

    (JMIR Medical Informatics, September 2024)

    Analysis of disease burden in patients with hereditary angioedema from Japan by patient-reported outcomes

    By Michihiro Hide, et al

    In a study conducted before the availability, in Japan, of long term prophylaxis for HAE, the authors aim to provide a benchmark of disease burden on Japanese patients. They concluded that weekly or monthly attack frequencies were associated with high disease burden and that regardless of attack frequency, patients reported fatigue, mood impairment, and depression.

    (The Journal of Dermatology, September 2024)

    Real-world outcomes of patients with hereditary angioedema with normal C1-inhibitor function and patients with idiopathic angioedema of unknown etiology in Canada

    By Adil Adatia, et al

    The authors investigated the characteristics and clinical outcomes of Canadian patients with HAE with normal C1-inhibitor. They suggest an unmet need for specific guidelines for these patients, and a better understanding of other underlying disease mechanisms is needed.

    (Allergy, Asthma and Clinical Immunology, September 2024)

    Long-term prevention of hereditary angioedema attacks with lanadelumab in adolescents

    By Timothy Craig, et al

    The authors investigated how well tolerated and effective the HAE medicine lanadelumab is in preventing HAE attacks in adolescents with HAE. The authors conclude that lanadelumab provided long-term efficacy in preventing HAE attacks, was associated with clinically meaningful improvements in health-related quality of life and high levels of treatment satisfaction, and was well-tolerated in adolescent patients.

    (Annals of Allergy, Asthma and Clinical Immunology, August 2024)

    Clinical profile and management of pediatric hereditary angioedema in resource-constrained settings: our experience from a single centre in North India

    By Ankur Kumar Jindal, et al

    The authors report from a single center in India that treats young patients with HAE. They conclude that this is one of the largest single-centre cohorts of pediatric HAE and the only one from resource-constrained settings. There were significant delays in diagnosis when the age of onset of symptoms was younger, and abdominal attacks were found to be less common in children as compared to adults.

    (Immunologic Research, September 2024)

    The complexities of decision-making associated with on-demand treatment of hereditary angioedema (HAE) attacks

    By Stephen D Betschel, et al

    This study aimed to determine patient behavior and the key factors that drive decision-making regarding using on-demand HAE treatment. The authors conclude that the burden of current treatments, such as the need for injections and infusions, may often cause delay, despite patients knowing this may lead to progression of HAE attacks

    (Allergy, Asthma and Clinical Immunology, July 2024)

    Initial experience of long-term prophylaxis with lanadelumab for hereditary angioedema in China: A clinical observation study on six patients

    By Wo Yao, et al

    The authors aimed to show the clinical efficacy and safety of using lanadelumab as long-term prophylaxis (LTP) in Chinese patients. These authors conclude that this is the first report of the clinical efficacy of lanadelumab and safety of LTP in HAE patients from the Chinese mainland. They believe the study demonstrates that a ‘reasonable dosage plan’ of lanadelumab can ensure quick and long-lasting protection from HAE attacks.

    (International Archives of Allergy and Immunology, October 2024)

    Selection of initiatives to improve the management of patients with hereditary angioedema by the hospital pharmacy using the nominal group technique

    By José Bruno Montoro Ronsano

    The authors aimed to identify and promote a range of initiatives within the hospital pharmacy that could improve the care of patients with HAE in Spain. The initiatives included how medicines are evaluated and selected, how pharmacies dispense medicines, follow-up, telemedicine consultations, patient education, and more joined-up working between other departments. The authors conclude that the implementation of such activities should enhance the way patients with HAE are cared for in Spain.

    (European Journal of Hospital Pharmacy, June 2024)

    Real-world outcomes in patients with hereditary angioedema prescribed lanadelumab versus other prophylaxis

    By John Anderson, et al

    Despite the availability of long-term prophylaxis in HAE, there is little real-world data on the use of these preventative treatments, according to the authors. They conclude that their data suggests lower attack frequency, lower symptomatic impact, and better quality of life in patients treated with lanadelumab than other prophylaxis in a real-world setting.

    (Allergy and Asthma Proceedings, September 2024)

    The Chronic Angioedema Registry (CARE): Rationale, methods and implementation

    By Thomas Buttgereit, et al

    The authors, including HAEi’s Anthony J. Castaldo and Henrik Balle Boysen, indicate that Chronic Angioedema Registry (CARE) is the first disease registry for recurrent angioedema of all presentations/aetiologies. The authors conclude that CARE will equip physicians with information to enhance patient care and guide future therapeutic decisions in all angioedemas, including HAE.

    (Clinical and Experimental Allergy, June 2024)

    A real-world study of hereditary angioedema patients due to C1 inhibitor deficiency treated with danazol in the Brazilian Public Health System

    By Alessandra Mileni Versuti Ritter, et al

    This study aimed to identify and characterize patients with HAE-C1-INH who used danazol prophylactic treatment in the Brazilian Public Health System (SUS) and the healthcare resource utilization (HCRU). The authors conclude that the high rate of attacks, hospitalizations, and general resource use in patients treated with danazol, highlights the necessity to increase awareness of new strategies and accurate approaches to treat HAE patients.

    (Frontiers in Medicine, September 2024)

    An international survey assessing the effects of the duration of attack-free period on health-related quality of life for patients with hereditary angioedema

    By Robbin Itzler, et al

    The authors examined whether there was any relationship between the amount of time people live attack-free and their quality of life. The authors conclude that their study showed that the longer patients are attack-free, the better their quality of life. They believe there are opportunities for new treatments to further increase the attack-free duration and improve quality of life for people with HAE.

    (Orphanet Journal of Rare Diseases, June 2024)

    Deciphering the landscape of hereditary angioedema in India: Perspective for Indian dermatologists

    By Prabal Barman, et al

    The authors tell us that this paper provides an update on HAE from a dermatologist’s perspective and the availability of first-line treatment options in India. Their conclusions are that:

    • HAE must be suspected in a patient presenting with non-itchy/non-urticarial episodic swelling.
    • Complement C4 is a simple and sensitive screening test for patients with HAE.
    • First-line treatment options for HAE are now available in India and must be used to avoid mortality and improve the quality of life.
    • Efforts should be continued to bring better treatment options for HAE.

    (Indian Journal of Dermatology, Venereology and Leprology, July 2024)

    Real-life experience of subcutaneous plasma derived C1-inhibitor as long-term prophylaxis in HAE-C1NH

    By Ana Entrala, et al

    The aim of their study was to assess efficacy and changes in health-related quality-of-life (HRQOL) in HAE-C1INH patients treated with SC pdC1INH (Berinert) as LTP under real-world conditions. The authors indicate that the majority of patients saw a reduction in attacks and improvements in their quality of life. The authors also note that using the Spanish Group for the Study of Bradykinin-mediated Angioedema (GEAB) protocol for starting long-term prophylaxis with subcutaneous plasma-derived C1-inhibitor proved useful for individualizing HAE treatment in our case series.

    (Journal of Investigational Allergology and Clinical Immunology, July 2024)

    Quantification of C1 inhibitor activity using a chromogenic automated assay: analytical and clinical performances

    By Yves Renaudineau, et al

    The authors found that an automated laboratory test for levels of functional C1 showed good performance, both at the analytical and diagnostic/clinical levels, which allowed its use in a clinical laboratory.

    (Clinical Chemistry and Laboratory Medicine, June 2024)

    Vascular endothelial growth factor (VEGF) emerging as a mediator of hereditary angioedema (HAE)

    By Pedro Giavina-Bianchi, et al

    Recognizing the ongoing advancements in our understanding of hereditary angioedema (HAE), the authors advocate for an improved classification system based on an understanding of endotypes, which are the underlying mechanisms of disease. The authors conclude that a precise and dynamic classification system for HAE that evolves with our growing understanding of the disease is needed. They argue that by adopting an endotype-based classification, we can more accurately reflect the underlying pathophysiological mechanisms, facilitating better patient care and more targeted treatments.

    (World Allergy Organization Journal, July 2024)

    Extension of the circulatory half-life of recombinant ecallantide via albumin fusion without loss of anti-kallikrein activity

    By Ghofran Al-Adimi, et al

    In this study of a new form of the HAE medicine ecallantide, the authors believed there is potential for the medicine to offer advances over current HAE treatment.

    (Journal of Biotechnology, August 2024)

    Subcutaneous C1 inhibitor for long-term prophylaxis in hereditary angioedema: A real-life experience

    By Krasimira Baynova, et al

    The authors report on 22 patients with C1-INH HAE who started subcutaneous C1-INH replacement treatment as long-term prophylaxis during the COVID-19 pandemic. The authors conclude that this treatment option proved to be an effective prophylactic treatment in a series of patients with HAE.

    (Journal of Investigational Allergology and Clinical Immunology, June 2024)

    Timing of onset of garadacimab for preventing hereditary angioedema attacks

    By Petra Staubach, et al

    In a letter to the Editor, the authors conclude that treatment with garadacimab results in early and durable protection against HAE attacks from Week 1 sustained to Month 6. The authors believe this contributes substantially towards achieving the primary goal of HAE treatment: complete disease control and normalization of patients’ lives. The authors say that protection against HAE attacks begins after the first administration of garadacimab, allowing clinicians to be confident in its treatment effect as early as Week 1 after treatment initiation.

    (Clinical and Experimental Allergy, October 2024)

News from the Industry

  • Astria reports Q2 financial results and provides a corporate update

    12 August, 2024

    Astria Therapeutics reports second quarter 2024 financial results and provides a corporate update

    Astra Therapeutics reported financial results for the second quarter ended 30 June 2024, and provided a corporate update.

    Jill C. Milne, PhD, Chief Executive Officer at Astria Therapeutics, said: “As we prepare for Phase 3 with initiation expected in Q1 2025, our vision for navenibart is to be the market-leading treatment for HAE, a large and growing market.”

    Navenibart (STAR-0215)

    • The World Health Organization’s (WHO) International Nonproprietary Names (INN) Expert Committee and the United States Adopted Names (USAN) Council of the American Medical Association (AMA) have adopted navenibart (nah-VEN-eh-bart) as the nonproprietary name for STAR-0215. Based on the WHO monoclonal antibody (mAb) nomenclature scheme which took effect in November 2021, the infix “-eni-” represents enzyme inhibitor and the suffix “-bart” represents a monoclonal antibody. The prefix “nav” represents Astria’s commitment to navigate the development navenibart with patients guiding the journey.
    • In March, the Company shared positive initial proof-of-concept results from the ALPHA-STAR Phase 1b/2 trial of navenibart in people with hereditary angioedema (HAE). Navenibart administered once or twice over six months reduced monthly attack rates by 90-96% and supports chronic dosing two or four times per year. Additionally, navenibart was well-tolerated with no serious adverse events and no discontinuations. The Company plans to report additional data from ALPHA-STAR in Q4 of 2024.
    • All of the original 16 target enrollment patients from ALPHA-STAR have entered or consented to enter the ALPHA-SOLAR long-term open-label trial. Initial safety and efficacy data from ALPHA-SOLAR, including from Q3M and Q6M administration, are expected mid-2025.
    • Based on the positive results from the ALPHA-STAR trial, Astria plans to advance navenibart to Phase 3 development. Pending regulatory feedback, the Company plans to initiate the Phase 3 program in Q1 2025 and expects top-line results by year-end 2026. The goal is to enable patients to choose what works best for them by developing both Q3M and Q6M dosing options. The company believes navenibart will be life-changing for HAE patients and has the opportunity to become the market leading HAE treatment.
    • In a separate press release, the company recently announced that it has chosen Ypsomed as its partner for the development of an autoinjector for navenibart.

    (Source: Astria)

  • Astria chooses Ypsomed’s YpsoMate as injection device for STAR-0215 for treatment of HAE

    12 August, 2024

    Astria Therapeutics chooses Ypsomed’s YpsoMate as the injection device for STAR-0215 for the treatment of hereditary angioedema

    Astra Therapeutics announced that it has chosen Ypsomed as its partner for the development of an autoinjector for STAR-0215.

    John Ruesch, Senior Vice President, Pharmaceutical Sciences and Technical Operations at Astria, said: “With STAR-0215’s profile, our goal is to develop a therapy that allows patients to choose an approach that works best for their lives with the option of infrequent three- and six-month administration. Our partnership with Ypsomed, a leading developer and manufacturer of injection systems, supports this goal through the planned development of an autoinjector that enables our vision for STAR-0215 to be a therapy that can effectively protect against HAE attacks while also having a very low burden of treatment and administration.”

    Assuming regulatory approval, Astria plans to launch STAR-0215 with both the Ypsomed YpsoMate autoinjector and a pre-filled syringe, which would allow patients to choose the administration regimen that would work best for their lives. Astria believes that an autoinjector option is a great choice for STAR-0215 due to its ease of use, needle shielding feature, and STAR-0215’s proprietary formulation that enables a quick injection with low risk of pain.

    The YpsoMate is a user-friendly two-step autoinjector. To inject, the cap is removed and then the device is firmly pushed on the patient’s skin. Visual and audible cues notify the patient of the start and end of the injection, and the needle is covered before and after the injection by a safety mechanism that protects against needle-stick injuries and ensures that patients do not see the needle. Furthermore, the autoinjector is based on Ypsomed’s carbon footprint reduction NetZero Program, which reflects a company-wide commitment to sustainability.

    (Source: Astria)

  • KalVista Announces Validation of MAA by the EMA for Sebetralstat for HAE

    15 August, 2024

    KalVista Announces Validation of Marketing Authorization Application by the European Medicines Agency for Sebetralstat for Hereditary Angioedema

    KalVista Pharmaceuticals announced that the European Medicines Agency (EMA) has validated the submission of a Marketing Authorization Application (MAA) for sebetralstat, a novel, investigational oral plasma kallikrein inhibitor for the on-demand treatment of hereditary angioedema (HAE).

    This application will now be reviewed by the EMA’s Committee for Medicinal Products for Human Use (CHMP) under the centralized licensing procedure for all 27 Member States of the European Union, as well as the EEA

    Ben Palleiko, Chief Executive Officer of KalVista, said: “The validation of this MAA, which we submitted in July, brings us another step closer to our goal of delivering sebetralstat on a global scale to people living with HAE. If approved, sebetralstat would be the first oral, on-demand treatment for HAE in Europe.”

    (Source: KalVista)

  • Pharvaris reports Q2 2024 financial results and provides business update

    16 August, 2024

    Pharvaris reports second quarter 2024 financial results and provides business update

    Pharvaris announced their financial results for the second quarter ended 30 June 2024, and provided a business update.

    Berndt Modig, Chief Executive Officer of Pharvaris, said: “Pharvaris supports the view of the HAE community that achievement of complete control of the disease and normalization of lives of people with HAE through long-term prophylaxis are the main goals of treatment in HAE. Obtaining alignment with regulatory agencies on our proposed global clinical development plan for deucrictibant as a prophylactic HAE treatment is an important milestone for the company.

    “RAPIDe-3 enrollment is progressing as planned, and CHAPTER-3 start-up activities are underway globally. Diligent execution of the RAPIDe-3 and the CHAPTER-3 pivotal clinical studies remains our top priority, with the goal of establishing differentiated data packages for deucrictibant in both on-demand and prophylaxis. Data from the ongoing open-label extensions in both on-demand and prophylaxis, as well as supplemental analyses from the RAPIDe-1 and CHAPTER-1 studies, will be presented at upcoming medical meetings. Pharvaris continues to operate from a strong financial position with a disciplined approach as we aspire to bring best-in-class oral therapies to the HAE community.”

    Recent Business Updates

    Alignment with regulatory authorities achieved regarding design of CHAPTER-3, a global Phase 3 study of deucrictibant for the prophylactic treatment of HAE. Pharvaris sought feedback and obtained alignment on key elements of a Phase 3 clinical study design during End-of-Phase 2 meetings with the US Food and Drug Administration (FDA), the European Union Committee for Medicinal Products for Human Use (CHMP), and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). CHAPTER-3 is planned as a randomized, double-blind, placebo-controlled Phase 3 study of orally administered deucrictibant extended-release tablets for the prophylactic treatment of HAE attacks. The study aims to enroll approximately 81 adult and adolescent participants (12 years and older) with HAE and randomize them in a 2:1 ratio to receive deucrictibant extended-release tablets (40 mg/day) or placebo once daily for 24 weeks. The primary endpoint of the study is to evaluate the efficacy of deucrictibant compared to placebo for prophylaxis against angioedema attacks as measured by the time-normalized number of investigator-confirmed HAE attacks during the 24-week treatment period. Other objectives of the study include evaluating additional clinically relevant outcomes, deucrictibant’s safety and tolerability, pharmacokinetics, and its impact on health-related quality of life in the prophylactic setting.

    Advancing RAPIDe-3 (NCT06343779), a global Phase 3 clinical study. RAPIDe-3, a global pivotal Phase 3 study of deucrictibant immediate-release capsules for the on-demand treatment of HAE attacks is progressing as planned with a target enrollment of approximately 120 participants. The primary efficacy endpoint is time to onset of symptom relief, as measured by Patient Global Impression of Change (PGI-C) rating of at least “a little better” for two consecutive timepoints within 12 hours post-treatment. Other efficacy endpoints include time to End of Progression (EoP) in attack symptoms, substantial symptom relief, complete attack resolution, and proportion of attacks achieving symptom resolution with one dose of deucrictibant as measured by Patient Global Impression of Severity (PGI-S) and by Angioedema Symptom Rating Scale (AMRA).

    Properties of deucrictibant further substantiated at recent medical congresses. Pharvaris presented data highlighting deucrictibant’s unique pharmacological and clinical properties at the CIIC Spring 2023 Conference, the 20th Annual Congress of International Drug Discovery Science and Technology (IDDST); the 2024 Eastern Allergy Conference (EAC); and the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2024. As part of the additional analyses presented at these congresses, one data highlight was a post-hoc analysis of the RAPIDe-1 data set which showed that 78.6% of the HAE attacks treated with deucrictibant in this study resolved within 24 hours.

    (Source: Pharvaris)

  • Deucrictibant clinical, real-World, nonclinical, and discovery data to be presented at the Bradykinin Symposium

    28 August, 2024

    Deucrictibant clinical, real-World, nonclinical, and discovery data to be presented at the Bradykinin Symposium

    Pharvaris announced the acceptance of two abstracts for oral presentation and ten abstracts for poster presentation at the upcoming 7th Bradykinin Symposium, to be held from 5-6 September 2024, in Berlin, Germany. Presentation details are as follows:

    • Title: Bradykinin Challenge Model in Humanized Bradykinin B2 receptor Transgenic Rat
      Presenter: Jolanta Skarbaliene PhD
      Format: Oral Presentation, Session II
      Date, time: Thursday 5 Sept, 15.05-15.15 CEST (9:05-9:15 am EDT)
    • Title: Prophylactic Treatment with Deucrictibant Improves HAE Disease Control and HRQoL
      Presenter: Markus Magerl MD
      Format: Oral Presentation, Session VII
      Date, time: Thursday 5 Sept, 15.05-15.15 CEST (9:05-9:15 am EDT)
    • Title: Long-Term Safety and Efficacy of Oral Deucrictibant for HAE Prophylaxis
      Presenter: Marc A. Riedl, MD MS
      Format: Poster Presentation
      Date, time: Friday 6 Sept, 15.35-16.15 CEST (9:35-10:15 am EDT)
    • Title: Treatment of HAE Attacks with Oral Deucrictibant: RAPIDe-2 Extension Results
      Presenter: Emel Aygören-Pürsün MD
      Format: Poster Presentation
      Date, time: Friday 6 Sept, 15.35-16.15 CEST (9:35-10:15 am EDT)
    • Title: Prophylaxis of Hereditary Angioedema Attacks with Oral Deucrictibant: CHAPTER-1 Results
      Presenter: Emel Aygören-Pürsün MD
      Format: Poster Presentation
      Date, time: Friday 6 Sept, 15.35-16.15 CEST (9:35-10:15 am EDT)
    • Title: Deucrictibant vs. Standard of Care in HAE: Propensity Score-Matched Analysis
      Presenter: Marc A. Riedl, MD MS
      Format: Poster Presentation
      Date, time: Friday 6 Sept, 15.35-16.15 CEST (9:35-10:15 am EDT)
    • Title: Clinical Trials Conformity with AURORA COS: a systematic literature review
      Presenter: Remy Petersen, MD PhD
      Format: Poster Presentation
      Date, time: Friday 6 Sept, 15.35-16.15 CEST (9:35-10:15 am EDT)
    • Title: Cardiovascular safety of repeated oral administration of the B2 receptor antagonist deucrictibant
      Presenter: Nieves Crespo PhD
      Format: Poster Presentation
      Date, time: Friday 6 Sept, 15.35-16.15 CEST (9:35-10:15 am EDT)
    • Title: The bradykinin challenge model translates across rat, monkey and human
      Presenter: Juan Bravo PhD
      Format: Poster Presentation
      Date, time: Friday 6 Sept, 15.35-16.15 CEST (9:35-10:15 am EDT)
    • Title: Deucrictibant inhibits carrageenan-induced edema in bradykinin B2 receptor transgenic rat
      Presenter: Anne Lesage PhD
      Format: Poster Presentation
      Date, time: Friday 6 Sept, 15.35-16.15 CEST (9:35-10:15 am EDT)
    • Title: A novel kinin biomarker assay for characterization of bradykinin-mediated disorders
      Presenter: Evangelia Pardali PhD
      Format: Poster Presentation
      Date, time: Friday 6 Sept, 15.35-16.15 CEST (9:35-10:15 am EDT)
    • Title: A HMWK capillary immunoblotting assay to characterize bradykinin-mediated disorders
      Presenter: Evangelia Pardali PhD
      Format: Poster Presentation
      Date, time: Friday 6 Sept, 15.35-16.15 CEST (9:35-10:15 am EDT)

    (Source: Pharvaris)

  • KalVista announces six abstracts accepted for presentation at the 2024 Bradykinin Symposium

    29 August, 2024

    KalVista Pharmaceuticals announces six abstracts accepted for presentation at the 2024 Bradykinin Symposium

    KalVista Pharmaceuticals today announced the acceptance of multiple abstracts at the 2024 Bradykinin Symposium taking place in Berlin, Germany on 5-6 September 2024.

    The following presentations will take place on Friday 6 September in the Kaiserin Friedrich-Haus Lecture Hall:

    Oral Presentations

    • Delayed On-demand Treatment of Hereditary Angioedema Attacks and Associated Barriers Reported by Italian patients: Mauro Cancian, Pietro Accardo, Francesco Arcoleo, Donatella Bignardi, Caterina Colangelo, Francesco Giardino, Antonio Gidaro, Marica Giliberti, Maria Domenica Guarino, Paola Lucia Minciullo, Stefania Nicola, Francesca Perego, Riccardo Senter, Giuseppe Spadaro, Paola Triggianese, Massimo Triggiani, Sherry Danese, Julie Ulloa, Vibha Desai, Tomas Andriotti, Paul Audhya, Andrea Zanichelli. Results shared in Session IV as an oral presentation and Q&A on Friday 6 September from 9:20 – 9:30 CET.
    • Pooled Sebetralstat Placebo-controlled Safety for On-demand Treatment of Hereditary Angioedema: Emel Aygören-Pürsün, Jonathan A. Bernstein, Danny Cohn, Henriette Farkas, William Lumry, Marc Riedl, Andrea Zanichelli, Samuel Owiredu-Yeboa, James Hao, Michael D. Smith, Christopher M. Yea, Paul K. Audhya, Marcus Maurer. Results shared in Session IV as an oral presentation and Q&A on Friday 6 September from 9:30 – 9:40 CET.
    • A Specific, Sensitivity Assay Measuring Patient Sample Plasma Kallikrein Activity: D. Lee, A. Ghannam, N. Murugesan, D. Vincent, A. Mogg, M. Smith, S. Hampton, E. Feener. Results shared in Session V as an oral presentation and Q&A on Friday 6 September from 11:50 – 12:00 CET.
    • KONFIDENT-S Interim Analysis: Sebetralstat for Hereditary Angioedema Attacks Including Laryngeal: Henriette Farkas, Marc A. Riedl, Emel Aygören-Pürsün, William R. Lumry, Andrea Zanichelli, James Hao, Matthew Iverson, Michael D. Smith, Christopher M. Yea, Paul K. Audhya, Jonathan A. Bernstein, Marcus Maurer, Danny M. Cohn. Results shared in Session VI as an oral presentation and Q&A on Friday 6 September from 13:10 – 13:20 CET.

    Poster Presentations

    • Pooled Sebetralstat Placebo-controlled Efficacy for On-demand Treatment of Hereditary Angioedema: Marcus Maurer, Jonathan A. Bernstein, Danny Cohn, Henriette Farkas, William Lumry, Marc Riedl, Andrea Zanichelli, James Hao, Matthew Iverson, Michael D. Smith, Christopher M. Yea, Paul K. Audhya, Emel Aygören-Pürsün. Results shared in a poster presentation and Q&A on Friday 6 September from 15:35 – 16:15 CET.
    • Phase 3 KONFIDENT Trial of Sebetralstat for HAE: European Subgroup: Andrea Zanichelli, Henriette Farkas, Emel Aygören-Pürsün, Fotios Psarros, Maria Staevska, Mauro Cancian, Sinisa Savic, Marcin Stobiecki, Eunice Dias de Castro, Marcin Kurowski, Ramon Lleonart, Vesna Panovska, Danny Cohn, Paul Audhya, Chris Yea, James Hao, Matthew Iverson, Michael Smith, Markus Magerl, Marcus Maurer. Results shared in a poster presentation and Q&A on Friday 6 September from 15:35 – 16:15 CET.

    (Source: KalVista)

  • Astria to present at upcoming Bradykinin Symposium

    30 August, 2024

    Astria to present at upcoming Bradykinin Symposium

    Astra Therapeutics announced that it will present two posters at the upcoming Bradykinin Symposium in Berlin, Germany on 6 September 2024:

    • Chris Morabito, M.D., Chief Medical Officer at Astria Therapeutics, will present information on ALPHA-SOLAR, a long-term open-label trial of navenibart (STAR-0215) in people living with HAE, in an encore presentation of a poster titled “Rationale and Design of the ALPHA-SOLAR Clinical Trial of STAR-0215.”
    • Kusumam Joseph, Senior Director of Medical Affairs at Astria Therapeutics, will present results from the Phase 1a trial of navenibart (STAR-0215) in an encore presentation of a poster titled “Phase 1a Trial of STAR-0215 for Hereditary Angioedema: Updated Results.”

    Both posters will be presented at the poster session on Friday 6 September at 3:35pm CEST.

    (Source: Astria)

  • KalVista announces FDA acceptance of NDA for sebetralstat for oral on-demand treatment of HAE

    3 September, 2024

    KalVista announces FDA acceptance of New Drug Application for sebetralstat for oral on-demand treatment of hereditary angioedema

    KalVista Pharmaceuticals announced that the US Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for sebetralstat, a novel, investigational oral plasma kallikrein inhibitor for the on-demand treatment of hereditary angioedema (HAE) attacks in adult and pediatric patients aged 12 years and older. The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of 17 June 2025. If approved, sebetralstat would be the first oral, on-demand treatment for HAE in adult and pediatric patients aged 12 years and older. The FDA is not currently planning to hold an advisory committee meeting to discuss the application.

    Ben Palleiko, Chief Executive Officer at KalVista, said: “We are thrilled with the FDA’s acceptance of our NDA for sebetralstat as it moves us one step closer to bringing a potentially transformative therapy to the HAE community. We understand that people living with HAE and their families carry a tremendous burden every day as they don’t know when the next attack may occur or if the attack could cause life-threatening consequences. The compelling data included in our NDA package show that sebetralstat has the potential to significantly alter the way people treat and manage their disease. Given that it could be the first, oral on-demand treatment for HAE, we continue to receive strong support and hear a sense of urgency among healthcare providers, advocates, patients and their families for sebetralstat. I am proud of the team at KalVista for their dedication to achieving this milestone and deeply grateful for the support of patients living with HAE, their families, the HAE scientific community, and the HAEA and HAEi patient advocacy organizations.”

    The NDA submission was supported by previously disclosed results, including data from the KONFIDENT phase 3 clinical trial and ongoing KONFIDENT-S open label extension trial. Sebetralstat met the primary endpoint for its phase 3 trial with both 300 mg and 600 mg formulations achieving the beginning of symptom relief significantly faster than placebo (p<0.0001 for 300 mg, p=0.0013 for 600 mg) and was well-tolerated, with a safety profile similar to placebo. In KONFIDENT-S, sebetralstat has enabled patients to treat attacks early with a median time from attack onset to treatment of 9 minutes, demonstrated a consistent safety and efficacy profile with KONFIDENT, and included a median time to beginning of symptom relief for laryngeal attacks of 1.3 hours.

    In addition to the NDA acceptance, KalVista recently announced that the European Medicines Agency (EMA) validated the submission of the Marketing Authorization Application (MAA) for sebetralstat. KalVista expects to file for approval in the UK, Japan, and other countries later in 2024.

    (Source: KalVista)

  • Pharvaris provides business update and expands development program for deucrictibant

    5 September, 2024

    Pharvaris provides business update and expands development program for deucrictibant

    Pharvaris today announced:

    • The planned initiation of CHAPTER-3, the pivotal Phase 3 study of deucrictibant extended-release tablets for the prophylactic treatment of HAE
    • Its intention to pursue clinical development of deucrictibant in a newly named indication, acquired angioedema due to C1-inhibitor deficiency (AAE-C1INH)
    • Presented a robust data set highlighting the differentiating characteristics of deucrictibant.

    Berndt Modig, Chief Executive Officer at Pharvaris, said: “Given the totality of data for deucrictibant, now bolstered by new data from ongoing long-term extension studies showing tolerability and efficacy in both prophylaxis and on-demand treatment, we believe deucrictibant has the potential to become a preferred therapy for the management of HAE. We remain focused on the efficient execution of our clinical studies, with the CHAPTER-3 study expected to initiate by the end of the year while RAPIDe-3 is progressing as planned. Pharvaris has the expertise to expand deucrictibant beyond HAE to other bradykinin-mediated-disease—such as AAE-C1INH—and we are excited to explore the potential for deucrictibant to meet a currently unaddressed medical need.”

    CHAPTER-3, a global, pivotal Phase 3 study of deucrictibant extended-release tablet for the prophylactic treatment of HAE attacks, is expected to initiate by year end 2024

    Start-up activities are on track to initiate CHAPTER-3 by the end of 2024. CHAPTER-3 will assess the efficacy and safety of once-daily dosing of the extended-release tablet formulation of deucrictibant, which is designed to provide sustained protection from HAE attacks by maintaining plasma exposure above therapeutic level for over 24 hours and achieving pharmacokinetic steady state in approximately two to three days.

    Stefan Abele PhD, Chief Technical Operations Officer of Pharvaris, commented: “Pharvaris’ supply chain and CMC teams have been working diligently to ensure timely delivery of deucrictibant extended-release tablets in the commercial formulation to our Phase 3 clinical sites. The use of deucrictibant extended-release tablets in the CHAPTER-3 Phase 3 study enables us to evaluate deucrictibant’s ability to address the need for improvements in quality-of-life that people living with HAE want and deserve: a therapy providing injectable-like efficacy, from the first day of therapy, with a favorable tolerability and the convenience of once-daily oral administration.”

    Pharvaris intends to pursue clinical development of deucrictibant in AAE-C1INH following publication of compelling data from an investigator-initiated trial

    Data in the Journal of Allergy and Clinical Immunology in July 2024 explored the potential for deucrictibant to address the unmet medical need for well-tolerated and effective therapies for the prophylactic and on-demand treatment of AAE-C1INH. A randomized, double-blind, placebo-controlled study was conducted by Investigators at the Amsterdam University Medical Center (Amsterdam UMC). Three people living with AAE-C1INH were enrolled; the individual mean monthly attack rates were 2.0, 0.6, and 1.0 during the placebo period and 0.0 across all participants during treatment with deucrictibant. There were no severe adverse events and one self-limiting treatment-emergent adverse event (abdominal pain).

    Remy S Petersen MD, of Amsterdam University Medical Center, stated: “There is an unmet need for therapies approved specifically for the treatment of AAE-C1INH. At Amsterdam UMC, we were pleased to confirm our hypothesis that a bradykinin B2 receptor antagonist, such as deucrictibant, has the potential to successfully prevent and treat AAE-C1INH. We look forward to continuing our collaboration with Pharvaris in the clinical development of deucrictibant for AAE-C1INH to further demonstrate the therapeutic benefit for those living with bradykinin-mediated angioedema.”

    Differentiated clinical profile of deucrictibant presented at the Bradykinin Symposium.

    A snapshot of long-term extension data from the ongoing prophylactic (CHAPTER-1 part 2) and on-demand (RAPIDe-2) extension studies provide evidence of the sustained product profile of deucrictibant in both HAE treatment settings.

    Upcoming Event: Consortium of Independent Immunology Clinics (CIIC)

    CIIC Fall 2024 Conference. Dallas, Texas, 13-14 September 2024. Two abstracts have been accepted for e-Poster presentation. Details are as follows:

    • Title: Long-Term Efficacy and Safety of Oral Deucrictibant, a Bradykinin B2 Receptor Antagonist, in Treatment of Hereditary Angioedema Attacks: Results of the RAPIDe-2 Extension Study

    Presenter: Joshua S. Jacobs MD

    Format: ePoster

    • Title: Long-Term Safety and Efficacy of Prophylactic Oral Deucrictibant, a Bradykinin B2 Receptor Antagonist, in Hereditary Angioedema: Results of the CHAPTER-1 Open Label Extension Study

    Format: Looped e-Poster Display Board

    (Source: Pharvaris)

  • Pharvaris presents deucrictibant long-term extension data for both the prophylactic and on-demand treatment of HAE at the Bradykinin Symposium 2024

    5 September, 2024

    Pharvaris presents deucrictibant long-term extension data for both the prophylactic and on-demand treatment of HAE at the Bradykinin Symposium 2024

    Pharvaris is highlighting the differentiated profile of deucrictibant as a prophylactic and on-demand treatment of HAE attacks at the Bradykinin Symposium 2024, being held in Berlin from 5-6 September 2024.

    Peng Lu MD PhD, Chief Medical Officer of Pharvaris, said: “Based on a snapshot analysis, treatment with deucrictibant led to a 93% reduction in attack rate compared to study baseline, a median attack rate of zero for every month, and a mean proportion of attack-free days of 99% after more than a year of mean duration of treatment in a prophylactic extension study. Together with the improvements in disease control and health-related quality of life observed in the randomized, placebo-controlled part of the CHAPTER-1 study, these data underscore the potential of deucrictibant to be an effective and well-tolerated prophylactic agent in the treatment of HAE.

    “Long-term extension data of deucrictibant in the on-demand setting similarly confirm its potential to become a preferred option for the treatment of HAE attacks with a median onset of symptom relief of 1.1 hours, as measured by Patient Global Impression of Change (PGI-C) and median complete resolution of 11.5 hours, as measured by Patient Global Impression of Severity (PGI-S). The rapid onset of symptom relief reported in RAPIDe-2 and the results of a propensity score-matched analysis favoring deucrictibant over standard of care provide confidence in our ability to differentiate deucrictibant in the on-demand HAE space. Lastly, the safety and tolerability profile of deucrictibant has been reaffirmed in multiple nonclinical and clinical studies.”

    Marc A Riedl MD MS, Professor of Medicine, Clinical Director of the U.S. Hereditary Angioedema Association (HAEA) Angioedema Center at the University of California San Diego (UCSD), Clinical Service Chief for Allergy/Immunology at UCSD, said: “The goal of HAE management is for affected individuals to live a normal life, ensuring they can engage in all work, school, family, and leisure activities as desired without limitation from angioedema symptoms. Therapies that offer improved efficacy, tolerability, and convenience have the potential to normalize the lives of people living with HAE. These long-term extension and health-related quality of life data, together with the Phase 2 clinical trial data, provide evidence of the benefits of deucrictibant as a potential treatment for HAE, and highlight the importance of additional data from late-stage clinical development of deucrictibant in both treatment settings.”

    (Source: Pharvaris)

  • Pharvaris presents data at the Bradykinin Symposium 2024

    5 September, 2024

    Pharvaris presents data at the Bradykinin Symposium 2024

    Pharvaris announced a summary of data being presented at the ongoing 7th Bradykinin Symposium. Details of the presentations are outlined below:

    Long-term safety and efficacy of oral deucrictibant for HAE prophylaxis

    A poster presentation by Marc A Riedl MD MS. In the current analysis of the ongoing open-label extension of the CHAPTER-1 Phase 2 study, deucrictibant 40 mg/day was well-tolerated, with no new safety signals observed. The results presented provide evidence of the long-term safety and efficacy of deucrictibant for the prevention of HAE attacks and support further development of deucrictibant as a potential prophylactic therapy for HAE. Results of this analysis provide support that:

    • Continuing deucrictibant treatment sustained the early-onset attack reduction seen in the randomized, placebo-controlled portion of the trial, with a median attack rate of zero for every month for over a year in the open-label part of the study
    • On average, less than one attack per year per participant was treated with on-demand medication

    Treatment of HAE attacks with oral deucrictibant: RAPIDe-2 extension results

    A poster presentation by Emel Aygören-Pürsün MD. In the current analysis of the ongoing RAPIDe-2 Phase 2/3 extension study, deucrictibant immediate release capsule was well-tolerated for all studied doses with no new safety signals observed. Results from the ongoing RAPIDe-2 extension are consistent with the randomized, placebo-controlled RAPIDe-1 Phase 2 study and provide evidence regarding the long-term safety and efficacy of deucrictibant IR capsule for repeat treatment of HAE attacks. Outcome analyses showed:

    • Median time to onset of symptom relief as measured by the Patient Global Impression of Change (PGI-C) was 1.1 hours, with 98.5% of attacks achieving onset of symptom relief by 12 hours
    • Median time to reduction in attack severity as measured by the Patient Global Impression of Severity (PGI-S) was 2.6 hours, with 97.7% of attacks achieving reduction in attack severity by 12 hours
    • Median time to complete attack resolution as measure by PGI-S was 11.5 hours, with 85.8% of attacks achieving complete attack resolution within 24 hours
    • Overall, 86.0% of attacks were treated with a single dose of deucrictibant immediate-release capsule

    Prophylactic treatment with deucrictibant improves HAE disease control and HRQoL

    An oral presentation by Markus Magerl MD. In the randomized, placebo-controlled part of CHAPTER-1, a Phase 2 clinical study of deucrictibant for the prophylactic treatment of HAE attacks, health-related quality of life was evaluated using several measures. In the study, it was demonstrated that deucrictibant treatment led to improvements in disease control versus placebo, with 90% of participants in the deucrictibant-groups demonstrating well-controlled HAE at week 12. Presentation details included:

    • Deucrictibant improved Angioedema Quality of Life Questionnaire (AE-QoL) scores, particularly in “functioning” and “fear/shame” domains compared to placebo
    • Deucrictibant-treated participants reported greater satisfaction than those treated with placebo with regards to effectiveness and the domain of global satisfaction, and a comparable satisfaction for side effects, as measured by Treatment Satisfaction Questionnaire for Medication (TSQM)

    Deucrictibant vs. standard of care in HAE: Propensity score-matched analysis

    A poster presentation by Marc A Riedl MD MS. A propensity score-matched comparison of clinical outcomes between a subgroup of attacks (N=73) from the RAPIDe-2 study and a subgroup of attacks (N=73) from an observational real-world study treated with standard of care, the outcomes were more favorable for the attacks treated with deucrictibant on PGI-C- and PGI-S-based assessments. Deucrictibant had a shorter (1.07 hours) median time to onset of symptom relief as measured by PGI-C “a little better” compared to standard of care (2.38 hours).

    Cardiovascular safety of repeated oral administration of the B2-receptor antagonist deucrictibant

    A poster presentation by Nieves Crespo PhD. In chronic nonclinical safety studies of deucrictibant in non-human primates, no evident effects on cardiac electrophysiology, morphology and hemodynamic parameters were observed. Deucrictibant has showed no evident effects on cardiac electrophysiology and hemodynamic parameters in clinical studies in humans to date, following prophylactic treatment up to 12 weeks of administration in the randomized, placebo-controlled part of the Phase 2 CHAPTER-1 clinical study and up to one year of mean duration of treatment in the ongoing open-label extension (OLE) part.

    Prophylaxis of hereditary angioedema attacks with oral deucrictibant: CHAPTER-1 results

    A poster presentation by Emel Aygören-Pürsün MD. The CHAPTER-1 study demonstrated that deucrictibant may significantly reduce the occurrence of HAE attacks, and clinically meaningful reduction in occurrence of moderate and severe HAE attacks, as well as HAE attacks treated with on-demand medication, was observed. CHAPTER-1 results provide evidence of the efficacy and safety of deucrictibant for the prevention of HAE attacks and support its further development as a potential prophylactic therapy for HAE.

    Clinical trials conformity with AURORA COS: a systematic literature review

    A poster presentation by Remy S Petersen MD. Conforming to a core outcome set (COS) across various study designs, such as the COS recommended for HAE clinical studies by the Panel of Experts participating in the AURORA Project, may homogenize the use of specific outcomes for clinical studies and support future indirect comparisons among interventions. The design of the RAPIDe-3 Phase 3 study of deucrictibant immediate-release capsule for the on-demand treatment of HAE attacks fully conforms with the AURORA COS based on its prespecified endpoints.

    Bradykinin challenge model in humanized bradykinin B2 receptor transgenic rat

    An oral presentation by Jolanta Skarbaliene PhD. The bradykinin (BK) challenge model is a tool to assess pharmacokinetic and pharmacodynamic activity of bradykinin B2 receptor antagonists. A BK challenge model was successfully developed in humanized bradykinin B2 receptor transgenic rats that are pharmacologically responsive to bradykinin B2 receptor antagonists. The BK challenge model in humanized bradykinin B2 receptor transgenic rats can offer a valuable, easy to manage, and cost-effective tool for efficacy studies compared to those involving non-human primates.

    Deucrictibant inhibits carrageenan-induced edema in bradykinin B2 receptor transgenic rat

    A poster presentation by Anne Lesage PhD. A humanized bradykinin B2 receptor transgenic rat model was used to address the challenge of deucrictibant species selectivity in experimental models. Oral deucrictibant inhibited carrageenan-induced paw edema in humanized bradykinin B2 receptor transgenic rats.

    The bradykinin challenge model translates across rat, monkey and human,

    A poster presentation by Juan Bravo PhD. The pharmacokinetics (PK) and pharmacodynamics (PD) of icatibant were analyzed from BK challenge studies in humanized bradykinin B2 receptor transgenic rats, non-human primates, and healthy volunteers. Analyses across species showed similar responses, demonstrating that the BK challenge model in transgenic rats and non-human primates may be predictive of PK/PD outcomes in humans.

    A novel kinin biomarker assay for characterization of bradykinin-mediated disorders

    A poster presentation by Evangelia Pardali, PhD. BK is involved in various physiological and pathological processes, including angioedema (AE). Differentiating BK-mediated from histamine-mediated AE and assessing the role of BK in the pathogenesis of other conditions by measuring kinin peptides remains a challenge due to their proteolytic instability and limitations of current analytical assays. A kinin biomarker assay was established and qualified, which could become a key tool for identifying, studying, and managing BK-mediated diseases.

    A HMWK capillary immunoblotting assay to characterize bradykinin-mediated disorders

    A poster presentation by Evangelia Pardali PhD. Activation of the plasma kallikrein-kinin system (KKS) can result in cleavage of high molecular weight kininogen (HMWK) and production of vasodilatory kinins, such as BK. A HMWK immunoblotting assay was established and qualified to reliably measure KKS biomarkers in human plasma and could become a key tool for identifying, studying, and managing BK-mediated diseases.

    (Source: Pharvaris)

  • KalVista reports first fiscal quarter results and provides operational update

    5 September, 2024

    KalVista Pharmaceuticals reports first fiscal quarter results and provides operational update

    KalVista Pharmaceuticals today provided an operational update and released financial results for the first fiscal quarter ended 31 July 2024.

    Ben Palleiko, CEO of KalVista, said: “We are excited about the steady progress we’ve made over the last few months including the most recent acceptance of our NDA by FDA and the EMA’s validation of our MAA.

    “We look forward to building on these recent milestones as we move toward filing for approval in the UK, Japan, and other countries later in 2024 and aim for our first commercial launch of sebetralstat in June 2025. I am grateful for the hard work of the entire KalVista team as we remain dedicated to getting this important treatment to people living with HAE.”

    Recent Business Highlights:

    Sebetralstat

    • In September, KalVista announced the US Food and Drug Administration (FDA) accepted its New Drug Application (NDA) for sebetralstat, a novel, investigational oral plasma kallikrein inhibitor for the on-demand treatment of hereditary angioedema (HAE) attacks in adult and pediatric patients aged 12 years and older. The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of 17June 2025.
    • Last month, the Company announced that the European Medicines Agency (EMA) validated the submission of the Marketing Authorization Application (MAA) for sebetralstat.
    • KalVista expects to file for approval in the UK, Japan, and other countries later in 2024. The Company has also engaged with the Access Consortium to maximize regulatory collaboration across countries and support a timely review process.
    • In June, ahead of schedule, KalVista initiated a pediatric clinical trial (KONFIDENT-KID) using an orally disintegrating tablet (ODT) formulation of sebetralstat designed for this population. KONFIDENT-KID will enroll approximately 24 children, with an age range of 2 to 11 years, across seven countries in North America, Europe and Asia. The trial has since started dosing patients and if approved, sebetralstat would be the first oral, on demand treatment for this population and only the second approved on-demand therapy of any type.
    • Data from the phase 3 KONFIDENT trial of sebetralstat was published in the New England Journal of Medicine (NEJM) and presented at the European Academy of Allergy and Clinical Immunology Congress 2024 (EAACI).

    (Source: KalVista)

  • BioCryst presents new Orladeyo (berotralstat) data at 7th Bradykinin Symposium

    6 September, 2024

    BioCryst presents new Orladeyo (berotralstat) data at 7th Bradykinin Symposium

    BioCryst Pharmaceuticals today announced the presentation of six posters, including the first interim real-word evidence from the APeX-N trial, and new data highlighting the value of shared decision making (SDM) between healthcare providers (HCPs) and their hereditary angioedema (HAE) patients to provide optimal patient outcomes.

    The company is presenting the posters at the 7th Bradykinin Symposium in Berlin from 5-6 September 2024.

    • APeX-N interim results: Oral berotralstat for HAE prophylaxis in Europe
    • HCP and patient perspectives: HAE long-term prophylaxis and shared decision-making
    • Adverse health outcomes and perspectives of androgen use in HAE
    • Evaluation of adherence to berotralstat in patients with hereditary angioedema
    • Tolerability and effectiveness of berotralstat for long-term prophylaxis in HAE
    • Effectiveness and safety of berotralstat in HAE with normal C1-inhibitor

    APeX-N interim results

    APeX-N is a European multi-center observational study assessing the safety (primary objective), effectiveness and quality of life (secondary objectives) of berotralstat 150 mg in routine clinical use. This interim analysis included 56 patients from the United Kingdom, France, Germany and Sweden.

    Non-serious gastrointestinal adverse events were reported in 12.5 percent of patients. Seven percent (n=4) of patients discontinued treatment (three due to unsatisfactory response, one to participate in a clinical trial). One patient had a severe HAE attack but continued treatment.

    Dr Sorena Kiani, Cnsultant Immunologist, at Royal Free London NHS Foundation, said: “These initial data from APeX-N in Europe reinforce and closely replicate the clinical trial and real-world evidence of berotralstat as the first oral prophylaxis for HAE.”

    HCP and patient perspectives: HAE long-term prophylaxis and shared decision-making

    This study, conducted in Germany, explored the dynamics between HCPs and patients in HAE management, identifying barriers to SDM and strategies to improve it. Ten HCPs participated in 60 minute interviews and simulated patient consultations. Eight HAE patients participated in 30 minute interviews. Participants then convened in structured focus groups to discuss their findings.

    The participants identified a need for enhanced HCP awareness of patient perspectives, more comprehensive HCP-patient conversations and improved education about HAE treatment management.

    Dr Emel Aygören-Pürsün, Department for Children and Adolescents, University Hospital Frankfurt, Germany, said: “The findings from this study support the need for further insights to develop future guidance and HAE management strategies to facilitate successful shared decision making and improved patient quality of life.”

    (Source: BioCryst)

  • KalVista Presents Sebetralstat Data at Bradykinin Symposium 2024

    6 September, 2024

    KalVista Pharmaceuticals Presents Sebetralstat Data at Bradykinin Symposium 2024

    KalVista Pharmaceuticals announced that it presented additional analyses of the efficacy and safety of sebetralstat from phase 2 and phase 3 double-blind, placebo-controlled crossover clinical trials as well as interim data from KONFIDENT-S, a phase 3 open-label extension trial, and real-world patient data at the Bradykinin Symposium 2024 taking place in Berlin, Germany, on 5-6 September 2024.

    Emel Aygören-Pürsün MD, Specialist in Internal Medicine at the Division of Oncology, Hematology and Hemostaseology at the Department for Children and Adolescents of the University Hospital Frankfurt, is a leading investigator for the phase 2, phase 3 KONFIDENT and KONFIDENT-S trials. She said: “Delay or denial in the treatment of HAE attacks is often related to the administration of the currently approved injectable on-demand treatments. The data presented today highlight that oral sebetralstat may remove these challenges and has a safety profile no different than placebo.”

    “Sebetralstat also resulted in rapid symptom relief in the clinical trials. If approved, sebetralstat might become a major advance for people living with HAE by addressing critical gaps in the current standard of care for on-demand treatment of attacks.”

    Paul Audhya MD MBA, Chief Medical Officer of KalVista Pharmaceuticals, said: “We are encouraged by the observation of exceptional consistency of both safety and efficacy across the entire clinical program for sebetralstat for the on-demand treatment of HAE. These data reinforce the potential for sebetralstat to transform the management of HAE.”

    The presentations at Bradykinin Symposium 2024 included:

    Delayed On-demand Treatment of Hereditary Angioedema Attacks and Associated Barriers Reported by Italian patients: Mauro Cancian, Azienda Ospedaliera, Università degli Studi di Padova, Padova, Italy (Oral presentation)

    Many patients did not meet guideline recommendations for early on-demand treatment following attack recognition, which resulted in more severe attacks.

    Pooled Sebetralstat Placebo-controlled Efficacy for On-demand Treatment of Hereditary Angioedema: Emel Aygören-Pürsün, University Hospital Frankfurt, Goethe University Frankfurt, Germany (Poster presentation)

    Pooled analysis of a larger number of HAE attacks corroborates the efficacy of sebetralstat for on-demand treatment, offering an oral administration route to potentially enable early treatment and rapid symptom relief.

    Pooled Sebetralstat Placebo-controlled Safety for On-demand Treatment of Hereditary Angioedema: Emel Aygören-Pürsün, University Hospital Frankfurt, Goethe University Frankfurt, Germany (Oral presentation)

    In the pooled safety analysis in phase 2 and phase 3 double-blind, placebo-controlled crossover trials, sebetralstat was well-tolerated with a safety profile no different than placebo.

    KONFIDENT-S Interim Analysis: Sebetralstat for Hereditary Angioedema Attacks Including Laryngeal: Henriette Farkas, Hungarian Angioedema Center of Reference and Excellence, Semmelweis University, Budapest, Hungary (Oral presentation)

    Among 640 attacks treated, median time to treatment was 9 minutes for all attacks and 8 minutes for laryngeal attacks; the median time to beginning of symptom relief was 1.8 hours for all attacks and 1.3 hours for laryngeal attacks.

    Phase 3 KONFIDENT Trial of Sebetralstat for HAE: European Subgroup: Andrea Zanichelli, Operative Unit of Medicine, Angioedema Center, IRCCS Policlinico San Donato, San Donato Milanese, Milan, Italy; Department of Biomedical Sciences for Health, University of Milan, Milan, Italy (Poster presentation)

    In the KONFIDENT phase 3 clinical trial, the positive efficacy and safety of sebetralstat as an on-demand treatment for HAE was consistent between European participants and the overall cohort.

    A specific, sensitivity assay measuring patient sample plasma kallikrein activity: Daniel Lee, KalVista Pharmaceuticals (Oral presentation)

    Measuring specific plasma kallikrein activity could be useful as a biomarker for normal C1INH, which currently has no standardized diagnostic pathway.

    (Source: KalVista)

  • Intellia to present data from the Phase 2 study of NTLA-2002 for the treatment of HAE at the 2024 ACAAI Annual Scientific Meeting

    12 September, 2024

    Intellia Therapeutics to present data from the Phase 2 study of NTLA-2002 for the treatment of hereditary angioedema (HAE) at the 2024 ACAAI Annual Scientific Meeting

    Intellia Therapeutics announced that data from the Phase 2 study of NTLA-2002 will be presented at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting, taking place 24-28 October 2024 in Boston, Massachusetts.

    Presentation Details:

    Title: Results From a Phase 2, Randomized, Placebo-Controlled Trial of CRISPR-Based Therapy NTLA-2002 for Hereditary Angioedema

    Session: Distinguished Industry & Late-breaking Oral Abstracts – Session 1

    Date and Time: Saturday 26 October 2024 from 4:30 – 6:30pm Eastern Time

    Presenter: Danny Cohn MD PhD, Internist, Department of Vascular Medicine, Amsterdam University Medical Center

    (Source: Intellia)

  • Astria to present at upcoming European Academy of Dermatology and Venerology Congress

    19 September, 2024

    Astria Therapeutics to present at upcoming European Academy of Dermatology and Venerology Congress

    Astra Therapeutics today announced that it will present initial navenibart ALPHA-STAR data at the upcoming European Academy of Dermatology and Venereology (EADV) Congress in Amsterdam, the Netherlands on 26 September 2024.

    Raffi Tachdjian, MD MPH, Associate Clinical Professor of Medicine and Pediatrics in the Division of Allergy and Clinical Immunology at the David Geffen School of Medicine at the University of California, Los Angeles (UCLA), will present a poster titled “ALPHA-STAR, a Phase 1b/2 Clinical Trial of Single and Multiple Doses of STAR-0215 in Patients with Hereditary Angioedema: Initial Safety and Efficacy Outcomes.”

    The poster will be presented in e-poster showcase six on Thursday 26 September 2024 at 4:20pm Central European Summer Time (CEST).

    (Source: Astria)

  • KalVista announces nine abstracts accepted for presentation at the ACARE Global Angioedema Forum 2024 (GAF)

    26 September, 2024

    KalVista Pharmaceuticals announces nine abstracts accepted for presentation at the ACARE Global Angioedema Forum 2024 (GAF)

    KalVista Pharmaceuticals today announced the acceptance of multiple abstracts at the ACARE Global Angioedema Forum 2024 (GAF) taking place in Copenhagen, Denmark from 4-5 October 2024.

    The following nine abstracts have been accepted for poster presentation on Friday 4 October between 6:00-7:00pm Central European Time:

    • Impact of Oral Sebetralstat on Anxiety Associated with Hereditary Angioedema Attacks in the Phase 3 KONFIDENT Trial: Marcus Maurer, Danny M. Cohn, Jonathan A. Bernstein, Henriette Farkas, William R. Lumry, Marc A. Riedl, Andrea Zanichelli, James Hao, Michael D. Smith, Paul K Audhya, Chris Yea, Emel Aygören-Pürsün
    • Anxiety Associated with Parenteral On-Demand Treatment for Hereditary Angioedema Attacks: Andrea Zanichelli, Pietro Accardo, Francesco Arcoleo, Donatella Bignardi, Caterina Colangelo, Francesco Giardino, Antonio Gidaro, Marica Giliberti, Maria Domenica Guarino, Paola Lucia Minciullo, Stefania Nicola, Francesca Perego, Riccardo Senter, Giuseppe Spadaro, Paola Triggianese, Massimo Triggiani, Sherry Danese, Julie Ulloa, Vibha Desai, Tomas Andriotti, Paul Audhya, Mauro Cancian
    • The Hereditary Angioedema (HAE) Attack Journey: A Conceptual Model of Patient Anxiety and On-Demand Treatment Burden During an HAE Attack: Douglas Jones, Hilary Longhurst, Mar Guilarte, Sally van Kooten, Neil Malloy, Markus Heckmann, Emily Carne
    • Impact of Delayed Treatment of Hereditary Angioedema Attacks on Quality of Life and Ability to Work: Patrick Yong, Rashmi Jain, Tomaz Garcez, Sorena Kiani-Alikhan, Vibha Desai, Tomas Andriotti, Paul Audhya, Sherry Danese, Julie Ulloa, Tariq El-Shanawany, Padmalal Gurugama, Sinisa Savic
    • Impact of Hereditary Angioedema Attacks on Quality of Life and Ability to Work Among UK Patients Receiving Long-term Prophylaxis or On-demand Treatment Only: Sinisa Savic, Tariq El-Shanawany, Padmalal Gurugama, Rashmi Jain, Vibha Desai, Tomas Andriotti, Paul Audhya, Sherry Danese, Julie Ulloa, Tomaz Garcez, Sorena Kiani-Alikhan, Patrick Yong
    • Phase 3 KONFIDENT Trial of Oral Sebetralstat for Treatment of Hereditary Angioedema Attacks: Analysis of the European and US Patient Subgroups: Andrea Zanichelli, Emel Aygören-Pürsün, Jonathan A Bernstein, Henriette Farkas, William R. Lumry, Marcus Maurer, Marc A Riedl, James Hao, Michael Smith, Paul Audhya, Chris Yea, Danny Cohn
    • Patient-Reported Benefits of Early On-demand Treatment of HAE Attacks: Mar Guilarte, Hilary Longhurst, Sally van Kooten, Neil Malloy, Markus Heckmann, Paula Busse
    • Treatment of HAE Attacks with Anticipated Future Oral On-demand Therapies as Reported by Patients: Anna Valerieva, Douglas Jones, Sally van Kooten, Neil Malloy, Markus Heckmann, Stephen Betschel
    • Global Frequency and Diagnosis of Hereditary Angioedema with Normal C1INH: A Real World ACARE Survey: Markus Magerl, Marc A Riedl, Sherry Danese, Julie Ulloa, Paul K Audhya, Marcus Maurer

    (Source: KalVista)

  • Astria to present at upcoming ACARE Global Angioedema Forum 2024

    27 September, 2024

    Astra Therapeutics announced that leading HAE experts will present two posters at the Global Angioedema Forum (GAF) in Copenhagen, Denmark on 4-5 October 2024.

    • Dr Aleena Banerji MD, Associate Professor of Medicine at Harvard Medical School and Clinical Director of the Allergy and Immunology Unit at Massachusetts General Hospital, will present information on ALPHA-SOLAR, a long-term open-label trial of navenibart (STAR-0215) in people living with HAE, in an encore presentation of a poster titled “Rationale and Design of the ALPHA-SOLAR Clinical Trial of STAR-0215.” The poster session will take place on Friday, 4 October at 6:00pm Central European Summer Time (CEST).
    • Dr William Lumry MD, Clinical Professor of Internal Medicine at the University of Texas Health Science Center at Dallas, will present results from the Phase 1b/2 trial of navenibart (STAR-0215) in an encore presentation of a poster titled “ALPHA-STAR, a Phase 1b/2 Clinical Trial of Single and Multiple Doses of STAR-0215 in Patients with Hereditary Angioedema: Initial Safety and Efficacy Outcomes.” The presentation will take place during a session titled “Emerging Treatment Options” on Saturday, 5 October at 9:30am CEST.

    (Source: Astria)

  • KalVista announces the submission of additional marketing authorization applications for Sebetralstat for the oral on-demand treatment of HAE

    30 September, 2024

    KalVista Pharmaceuticals today announced Marketing Authorization Application (MAA) submissions to the regulatory authorities in the United Kingdom, Switzerland, Australia, and Singapore for sebetralstat, a novel, investigational oral plasma kallikrein inhibitor for the on-demand treatment of hereditary angioedema (HAE) attacks in adults and adolescents aged 12 years and older. The four MAAs have been submitted via the Access Consortium framework for which KalVista has obtained a four-way work-sharing agreement by the Medicines and Healthcare product Regulatory Agency, Swissmedic, the Therapeutic Goods Administration and Health Sciences Authority. The Access Consortium is designed to maximize regulatory collaboration across countries and support a timely review process.

    Ben Palleiko, CEO of KalVista, said: “Today’s news, which comes only a few weeks following our announcements regarding our US FDA PDUFA date of June 17 and validation of our MAA by EMA, further underscores our focus and dedication to getting sebetralstat to as many people living with HAE as possible. To serve that goal, we are building a global commercial presence to provide the greatest possible access to potentially the first oral on-demand treatment for this debilitating disease. I am proud of the KalVista team for their dedication and continued hard work submitting these additional MAAs.”

    The MAA submissions are supported by previously disclosed results, including data from the KONFIDENT phase 3 clinical trial and ongoing KONFIDENT-S open label extension trial. Sebetralstat met the primary endpoint for its phase 3 trial with both 300 mg and 600 mg formulations achieving the beginning of symptom relief significantly faster than placebo (p<0.0001 for 300 mg, p=0.0013 for 600 mg) and was well-tolerated, with a safety profile similar to placebo. In KONFIDENT-S, sebetralstat has enabled patients to treat attacks early with a median time from attack onset to treatment of 9 minutes, demonstrated a consistent safety and efficacy profile with KONFIDENT, and included a median time to beginning of symptom relief for laryngeal attacks of 1.3 hours. KONFIDENT and KONFIDENT-S are the only clinical trials ever conducted in HAE that instruct participants to treat their attacks as early as possible, regardless of severity, in accordance with on-demand treatment guidelines. Early treatment of attacks, prior to progression, is a critical element in proper management of HAE, to minimize symptom burden.

    (Source: KalVista)

  • Astria receives FDA orphan drug designation for navenibart (STAR-0215) for the treatment of HAE

    30 September, 2024

    Astria receives FDA orphan drug designation for navenibart (STAR-0215) for the treatment of HAE

    Astra Therapeutics announced that navenibart (STAR-0215) has been granted Orphan Drug Designation for the treatment of hereditary angioedema (HAE) by the US Food and Drug Administration (FDA). Navenibart is a monoclonal antibody inhibitor of plasma kallikrein designed to provide long-acting attack prevention for HAE. Initial results from the Phase 1b/2 ALPHA-STAR clinical trial of navenibart have demonstrated a favorable safety and tolerability profile and a reduction of monthly attack rates by 90-96% when dosed once or twice over six months.

    Jill C Milne PhD, Chief Executive Officer of KalVista, said: “Receiving orphan drug designation for navenibart is an important affirmation of our belief that there is a significant unmet need for people living with HAE. We expect to share additional results from the ALPHA-STAR trial in Q4 and to progress navenibart into a Phase 3 trial initiating in the first quarter of 2025.”

    The FDA’s Orphan Drug Designation program grants orphan status to medicines intended for the safe and effective prevention, diagnosis, or treatment of rare diseases or disorders that affect fewer than 200,000 people in the United States. Orphan status provides sponsors with development and commercial incentives for designated compounds and medicines.

    (Source: Astria)

  • KalVista presents sebetralstat data at the ACARE Global Angioedema Forum 2024

    4 October, 2024

    KalVista Pharmaceuticals announced that it presented data showing the effectiveness of sebetralstat in reducing anxiety among people experiencing hereditary angioedema (HAE) attacks at the HAEi Global Angioedema Forum (GAF) taking place in Copenhagen, Denmark from 4-5 October 2024. These data were generated in the KONFIDENT phase 3 clinical trial, for which the Company disclosed top line results in February 2024. Sebetralstat is a novel, investigational oral plasma kallikrein inhibitor for the on-demand treatment of HAE attacks in adults and adolescents aged 12 years and older.

    William Lumry MD, of Allergy and Asthma Research Associates in the United States, said: “The data presented at the Global Angioedema Forum highlights that people living with HAE often experience substantial anxiety when they have attacks, which reduces quality of life. In KONFIDENT, oral sebetralstat significantly reduced anxiety during attacks compared with placebo, especially among participants with moderate-to-extreme anxiety. If approved, sebetralstat has the potential to become an important option for people living with HAE who experience attack-related anxiety, enabling them to treat early and recover from their attacks sooner.”

    Additional poster presentations at the HAEi Global Angioedema Forum 2024:

    • Anxiety Associated with Parenteral On-Demand Treatment for Hereditary Angioedema Attacks: Riccardo Senter, Azienda Ospedaliera, Università degli Studi di Padova, Italy
    • The Hereditary Angioedema (HAE) Attack Journey: A Conceptual Model of Patient Anxiety and On-Demand Treatment Burden During an HAE Attack: Douglas Jones, Metrodora Institute, Salt Lake City, United States
    • Impact of Delayed Treatment of Hereditary Angioedema Attacks on Quality of Life and Ability to Work: Patrick Yong, Frimley Health NHS Foundation Trust, United Kingdom
    • Impact of Hereditary Angioedema Attacks on Quality of Life and Ability to Work Among UK Patients Receiving Long-term Prophylaxis or On-demand Treatment Only: Patrick Yong, Frimley Health NHS Foundation Trust, United Kingdom
    • Phase 3 KONFIDENT Trial of Oral Sebetralstat for Treatment of Hereditary Angioedema Attacks: Analysis of the European and US Patient Subgroups: Andrea Zanichelli, Operative Unit of Medicine, Angioedema Center, IRCCS Policlinico San Donato, San Donato Milanese, Milan, Italy; Department of Biomedical Sciences for Health, University of Milan, Italy (Poster presentation)
    • Patient-Reported Benefits of Early On-demand Treatment of HAE Attacks: Mar Guilarte, Allergy Department, Hospital Universitari Vall d’Hebron, Spain
    • Treatment of HAE Attacks with Anticipated Future Oral On-demand Therapies as Reported by Patients: Anna Valerieva, Department of Allergology, Medical University of Sofia, Bulgaria
    • Global Frequency and Diagnosis of Hereditary Angioedema with Normal C1INH: A Real World ACARE Survey: Markus Magerl, Angioedema Center of Reference and Excellence (ACARE), Institute of Allergology, Charité – Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Germany; Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Immunology and Allergology, Germany

    In Memoriam: KalVista would like to acknowledge and honor the memory of Prof. Marcus Maurer, Professor of Dermatology and Allergy, Executive Director of the Institute of Allergology at the Charité – Universitätsmedizin Berlin, and Co-Director of Allergology and Immunology at the Fraunhofer Institute for Translational Medicine and Pharmacology ITMP. Our thoughts remain with his family and the entire angioedema and urticaria community.

    (Source: KalVista)

  • Intellia announces initiation of HAELO Phase 3 study of NTLA-2002, an investigational in vivo CRISPR gene editing treatment for HAE

    7 October, 2024

    Intellia Therapeutics announced the initiation of HAELO, a global, pivotal Phase 3 study of NTLA-2002 for the treatment of hereditary angioedema (HAE). NTLA-2002 is a wholly owned investigational in vivo CRISPR-based gene editing therapy in development as a single-dose treatment for this potentially life-threatening disease.

    Patient screening is active following Intellia’s successful end-of-Phase 2 meeting and submission of an Investigational New Drug Application amendment to the U.S. Food and Drug Administration (FDA).announced that data from the Phase 2 study of NTLA-2002 will be presented at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting, taking place 24-28 October 2024 in Boston, Massachusetts.

    Intellia President and Chief Executive Officer, John Leonard MD, said: “Initiation of the HAELO Phase 3 trial is a significant milestone for Intellia as we enter the final stage of clinical development for NTLA-2002 for people living with hereditary angioedema. Data from the ongoing Phase 1/2 study showed great promise that a single-dose treatment can lead to a complete response – no more attacks and no further treatment required. We are working urgently to bring forward NTLA-2002 to address the real-world needs of people suffering from this disease and, ultimately, believe it will bring significant value to patients, physicians and payors.”

    HAELO is a global, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of NTLA-2002 in 60 adults with Type I or Type II HAE. Patients will be randomized 2:1 to receive a single 50 mg infusion of NTLA-2002 or placebo. Patients randomized to the placebo arm will be eligible for optional crossover to NTLA-2002 at week 28. The primary endpoint is the change in number of HAE attacks from week 5 through week 28.

    Intellia is initiating the Phase 3 study based on positive safety and efficacy data from the ongoing Phase 1/2 study (NCT05120830) of NTLA-2002. Interim Phase 1 clinical data showed dramatic reductions in attack rate, as well as consistent, deep and durable reductions in kallikrein levels. Intellia previously announced positive toplines results from the Phase 2 portion of the study. The Company plans to present the detailed results at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting, taking place October 24 – 28 in Boston, Massachusetts.

    (Source: Intellia)

  • BioCryst to Present New Real-World Evidence on Long-Term Prophylaxis in HAE, including Orladeyo (berotralstat), at the ACAAI 2024 Annual Scientific Meeting

    10 October, 2024

    BioCryst to Present New Real-World Evidence on Long-Term Prophylaxis in HAE, including Orladeyo (berotralstat), at the American College of Allergy, Asthma & Immunology 2024 Annual Scientific Meeting

    BioCryst Pharmaceuticals announced that the company will present four abstracts on oral, once-daily Orladeyo (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE) in patients 12 years and older at the Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI). The meeting will take place in Boston from October 24-28, 2024.

    Poster Presentations

    • Adherence And Persistence Among Hereditary Angioedema Patients Treated With Berotralstat, Lanadelumab, And Subcutaneous Plasma-Derived C1-Inhibitor; ePoster #R072; 2:30-2:45pm Eastern Time (ET), Friday 25 October 2024; Monitor #19, Hall A
    • Sustained Real-World Attack Reductions Following Berotralstat Initiation Among Patients with Hereditary Angioedema with C1-Inhibitor Deficiency; ePoster #R092; 3:30-3:45pm ET, Friday 25 October 25; Monitor #20, Hall A
    • Sustained Real-World Attack Reductions Following Berotralstat Initiation Among Patients with Hereditary Angioedema without C1-Inhibitor Deficiency; ePoster #R093; 3:45-4:00 pm ET, Friday 25 October; Monitor #20, Hall A
    • Long-Term Prophylactic Treatment Preferences of Patients With Hereditary Angioedema; ePoster #R081; 4:45-5:00pm ET, Friday 25 October; Monitor #19, Hall A

    (Source: BioCryst)

  • BioCryst to present new real-world evidence showing significant reductions in medical visits and hospitalizations after starting Orladeyo (berotralstat)

    14 October, 2024

    BioCryst to present new real-world evidence showing significant reductions in medical visits and hospitalizations after starting Orladeyo (berotralstat)

    BioCryst Pharmaceuticals announced new real-world evidence on the use of oral, once-daily Orladeyo (berotralstat) demonstrating that patients with hereditary angioedema (HAE) in the United States experience significant reductions in healthcare resource utilization (HRU), including significant reductions in hospitalizations, emergency room visits and use of on-demand therapies, after beginning treatment with Orladeyo.

    The study will be presented in a poster at the Academy of Managed Care Pharmacy (AMCP) Nexus 2024 national meeting, which is being held in Las Vegas from 14-17 October 2024.

    Professor Sandra Christiansen MD, Professor of Medicine and Director of Translational Research at the US HAEA Angioedema Center at the University of California, San Diego, said: “Our interrogation of claims data builds upon previously reported findings demonstrating that Orladeyo not only has a favorable impact on the lives of patients with HAE – but the broader healthcare system, as well.

    “Here, our analysis reveals that significant reductions in healthcare resource utilization are achieved across multiple outcomes – such as reductions in hospitalizations and medical visits, including those related to HAE attacks. Decreases in on-demand treatment were also observed. Taken together, our investigation provides promising real-world evidence which supports Orladeyo’s clinical and financial value as a prophylactic therapy for HAE.”

    (Source: BioCryst)

  • EC grants orphan medicinal product designation for navenibart, Astria’s investigational therapy for the treatment of HAE

    16 October, 2024

    European Commission grants orphan medicinal product designation for navenibart, Astria Therapeutics’ investigational therapy for the treatment of hereditary angioedema

    Astra Therapeutics announced that the European Commission (EC) has granted Orphan Medicinal Product Designation (OMPD) to navenibart for the treatment of hereditary angioedema (HAE). Navenibart is a monoclonal antibody inhibitor of plasma kallikrein designed to provide long-acting attack prevention for HAE.

    Initial results from the Phase 1b/2 ALPHA-STAR clinical trial of navenibart have demonstrated a favorable safety and tolerability profile and a reduction of monthly attack rates by 90-96% when dosed once or twice over six months.

    Jill C Milne PhD, Chief Executive Officer of Astria, said: “We are very pleased to receive Orphan Medicinal Product Designation for navenibart in Europe,” said. We believe that OMPD status, in combination with the FDA’s designation of navenibart as an orphan drug in the United States, reinforces both the global unmet need for people with HAE and the potential for navenibart to change the way that people live with HAE. We look forward to the expected progression of navenibart into a Phase 3 trial in the first quarter of 2025 and continuing our efforts to bring a new treatment option to people living with HAE around the world.”

    In the European Union (EU), OMPD is granted by the EC following a recommendation from the European Medicines Agency (EMA) via its Committee for Orphan Medicinal Products (COMP). OMPD status provides incentives for drug development sponsors to develop products for diagnosing, preventing or treating life-threatening or very serious conditions that are rare and affect not more than five in 10,000 persons in the EU. Sponsors may receive regulatory and financial incentives as well as eligibility for EMA assistance in optimizing the candidate’s clinical development.

    (Source: Astria)

  • KalVista to present data at the 2024 ACAAI Annual Scientific Meeting

    18 October, 2024

    KalVista Pharmaceuticals to present data at the 2024 Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI)

    KalVista Pharmaceuticals announced that five abstracts have been accepted for e-Poster presentation at the 2024 Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI), taking place in Boston, MA from October 24-28. Presentations include:

    • Patient-Reported Anxiety Impacts Utilization of Injectable On-demand Treatment of Hereditary Angioedema Attacks: Cristine Radojicic, Autumn Burnette, Sally van Kooten, Neil Malloy, Markus Heckmann, Hilary Longhurst. Results shared as an e-Poster Presentation on Friday 25 October at 2:00pm Eastern Time (ET), Monitor 20, Exhibit Hall A.
    • On-demand Treatment of Laryngeal Hereditary Angioedema Attacks with Sebetralstat: Pooled Analysis from KONFIDENT and KONFIDENT-S: Emel Aygören-Pürsün, Jonathan A Bernstein, William R Lumry, Paul K Audhya, James Hao, Michael D Smith, Christopher M Yea, Marc A Riedl. Results shared as an e-Poster Presentation on Friday 25 October at 5:00pm ET, Monitor 20, Exhibit Hall A.
    • Substantial Reduction of Hereditary Angioedema Attack Symptom Burden in the Sebetralstat Phase 3 KONFIDENT Trial: William R Lumry, Danny M Cohn, Jonathan A Bernstein, Paul K Audhya, James Hao, Michael D Smith, Christopher M Yea, Marc A Riedl. Results shared as an e-Poster Presentation on Friday 25 October at 5:00pm ET, Monitor 19, Exhibit Hall A.
    • Indirect Treatment Comparison of Oral Sebetralstat and Intravenous Rhc1-INH as On-Demand Treatments for Hereditary Angioedema: H Henry Li, Markus Magerl, Timothy Craig, Michael E Manning, Noemi Hummel, Alice Wang, Paul K Audhya, Jonathan A Bernstein. Results shared as an e-Poster Presentation on Friday 25 October at 5:15pm ET, Monitor 19, Exhibit Hall A.
    • Correlation of Time to Treatment with Attack Duration in the Sebetralstat KONFIDENT Phase 3 Trial: Timothy J Craig, Jonathan A Bernstein, Hilary Longhurst, James Hao, Michael D Smith, Paul K Audhya, Christopher M Yea, Marcus Maurer. Results shared as an e-Poster Presentation on Friday 25 October at 5:30pm ET, Monitor 19, Exhibit Hall A.

    (Source: KalVista)

  • Astria to present at upcoming ACAAI Annual Scientific Meeting

    21 October, 2024

    Astria Therapeutics to present at upcoming American College of Allergy, Asthma, and Immunology Annual Scientific Meeting

    Astra Therapeutics announced that leading HAE experts will present two posters at the American College of Allergy, Asthma, and Immunology (ACAAI) Annual Scientific Meeting in Boston, Massachusetts on 25 October 2024.

    Dr Aleena Banerji MD, Associate Professor of Medicine at Harvard Medical School and Clinical Director of the Allergy and Immunology Unit at Massachusetts General Hospital, will present new quality of life data from initial results from the Phase 1b/2 trial of navenibart (STAR-0215) in a presentation of a poster titled “STAR-0215 Induces Rapid Improvements of Quality of Life in HAE Patients in the ALPHA-STAR Trial.” The presentation will take place in Exhibit Hall A on Friday 25 October at 3:30pm Eastern Standard Time (EST).

    Astria sponsored additional quality of life research in people living with HAE that will be presented by Dr Donald Levy MD, Health Sciences Professor of Medicine in the Division of Basic and Clinical Immunology at the University of California at Irvine. Dr Levy will present a poster titled “Quality of Life and Burden of Disease in Patients with Hereditary Angioedema and their Caregivers” in Exhibit Hall A on Friday 25 October at 3:45pm EST.

    (Source: Astria)

  • Intellia presents positive results from the Phase 2 Study of NTLA-2002

    24 October, 2024

    Intellia presents positive results from the Phase 2 Study of NTLA-2002, an investigational in vivo crispr gene editing treatment for hereditary angioedema (HAE)

    Intellia Therapeutics announced positive Phase 2 data from the ongoing Phase 1/2 study of NTLA-2002 in patients with hereditary angioedema (HAE), with results continuing to indicate that NTLA-2002 has the potential to eliminate HAE attacks following a one-time infusion.

    Results were published in The New England Journal of Medicine and will be presented on Saturday 26 October at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Scientific Meeting in Boston, Massachusetts.

    Intellia President and Chief Executive Officer John Leonard MD, said: “These positive NTLA-2002 Phase 2 results underscore the tremendous potential of our in vivo CRISPR gene editing therapy to be a functional cure and redefine the treatment paradigm for HAE. The Phase 2 data demonstrated that a majority of patients in the 50mg arm experienced a complete response — no attacks at all and no further treatment needed — after a one-time infusion of NTLA-2002 through the latest follow-up, consistent with the long-term Phase 1 data. We are highly encouraged by these results, which we believe sets NTLA-2002 apart from other prophylaxis treatments. What was previously an unimaginable potential to be free of chronic therapy is one step closer to becoming a reality for the HAE community.”

    Danny Cohn MD PhD, Internist, Department of Vascular Medicine, Amsterdam University Medical Center and the Phase 2 study’s lead principal investigator, said: “Approved HAE therapies can reduce but frequently do not eliminate all angioedema attacks and require chronic administration, resulting in a significant treatment burden and a major impact on the quality of life for people living with HAE. These NTLA-2002 Phase 2 data are remarkable, showing this investigational therapy could permanently stop swelling attacks with a single infusion. I am optimistic that NTLA-2002 will change the way we treat HAE and put an end to the need for a lifetime of chronic treatment.”

    (Source: Intellia)

  • BioCryst presents new real-world evidence showing high adherence and persistence rates with Orladeyo (berotralstat)

    24 October, 2024

    BioCryst presents new real-world evidence showing high adherence and persistence rates with Orladeyo (berotralstat)

    BioCryst Pharmaceuticals announced new real-world comparative research on the use of oral, once-daily Orladeyo (berotralstat) that found high rates of adherence and persistence for Orladeyo, similar to the rates observed with two other long-term prophylactic (LTP) therapies for HAE.

    The company also announced new real-world evidence showing statistically significant and sustained HAE attack rate reductions after initiating Orladeyo in patients with HAE, regardless of their C1-inhibitor (C1-INH) deficiency status, and new findings from an HAE patient survey confirming patient preference for an oral LTP therapy.

    Dr Donald S Fong, Chief Medical Officer of BioCryst, said: “These results build on previous research that highlights how the real-world effectiveness and convenience of our oral, once-daily prophylactic therapy are differentiators for patients. The strong adherence, persistence and preference for Orladeyo we report here provide further evidence that enables physicians to optimize individualized treatment recommendations for each of their patients. We continue to generate real-world evidence demonstrating that Orladeyo is a powerful, potentially life-changing treatment for many people with HAE.”

    The data are being presented at the Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI), which is taking place in Boston from 24-28 October 2024.

    (Source: BioCryst)

  • Pharvaris presents clinical and non-clinical data supporting HAE development program at the 2024 ACAAI Annual Scientific Meeting

    24 October, 2024

    Pharvaris presents clinical and non-clinical data supporting HAE development program at the 2024 ACAAI Annual Scientific Meeting

    Pharvaris announced data from seven posters that will be presented at the 2024 Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI).

    Pharvaris mourns our distinguished colleague, Prof. Marcus Maurer, who passed away during the development and finalization of these posters.

    Peng Lu MD PhD, Chief Medical Officer of Pharvaris, said: “The totality of data supporting deucrictibant’s potential best-in-class profile in both prophylaxis and on-demand remains consistent and provides compelling validation for its continued clinical development. The data presented at ACAAI continue to clinically derisk deucrictibant and contribute to excitement for deucrictibant to become a preferred therapy in HAE.”

    The CHAPTER-1 Phase 2 data provide proof of the efficacy and safety of deucrictibant for the prevention of HAE attacks and support its further development as a potential prophylactic therapy for HAE, which will be presented by H James Wedner MD. Specifically, Dr Wedner explores deucrictibant’s ability to decrease the median percentage of days with symptoms to 1.7% (40 mg/day) from 14.6% (placebo).

    Results of the ongoing CHAPTER-1 open-label extension study provide further evidence on the long-term safety and efficacy of deucrictibant for prevention of HAE attacks and support further development of deucrictibant as a potential prophylactic therapy for HAE, which will be presented by John Anderson MD. Importantly, approximately 80% of participants achieved at least a 90% reduction in attack rate relative to the study baseline (as studied in the randomized clinical trial).

    Pharvaris’ confidence in the design of both the prophylactic and on-demand Phase 2 and Phase 3 studies is supported by nonclinical data in the bradykinin (BK) challenge model in non-human primates (NHPs). The pharmacokinetic and pharmacodynamic (PK/PD) profile of deucrictibant in the NHP BK challenge was shown to be predictive of the human PK/PD in the human BK challenge, as presented by Juan Bravo PhD. Successful predictions of efficacious dosing of deucrictibant in humans were obtained following in-house modeling of nonclinical. in line with the Phase 2 clinical studies of deucrictibant for both the prophylactic and the on-demand treatment of HAE attacks.

    (Source: Pharvaris)

  • Astria to present at upcoming CSACI Annual Scientific Meeting

    30 October, 2024

    Astria Therapeutics to present at upcoming Canadian Society of Allergy and Clinical Immunology Annual Scientific Meeting

    Astra Therapeutics announced that it will present interim quality of life results from the ALPHA-STAR trial of navenibart (STAR-0215) at the Canadian Society of Allergy and Clinical Immunology (CSACI) Annual Scientific Meeting in Banff, Canada on 7 November 2024.

    Adil Adatia MD, Assistant Professor in the Department of Medicine at the University of Alberta in Edmonton, Alberta and Director of the University of Alberta Angioedema Centre of Reference and Excellence (ACARE), will present a poster titled: Quality of life (QoL) improvements in hereditary angioedema (HAE) with STAR-0215; interim results from the phase 1b/2 ALPHA-STAR clinical trial. The poster will be exhibited in the Van Horne Foyer from 10:00am Mountain Daylight Time on Thursday 7 November until the end of day on Saturday 9 November 2024.

    (Source: Astria)

HAEi Around the World

Currently, there are HAE Member Organizations in 101 countries. You will find a great deal of vital information on the HAE representations around the globe at haei.org, and the world map will provide you with contact information for the Member Organizations, ACARE centers, hospitals, physicians, and available medication.

The information on haei.org is updated as soon as HAEi receives fresh data from the national Member Organizations.

Global Perspectives · Issue 3/2024 · November 2024

Magazine Staff: Henrik Balle Boysen, President; Deborah Corcoran, Chief Scientific Officer; Stuart Mayell, Editor in Chief, Global Perspectives; Rikke Sørensen, Manager, Communication, Design, and Graphics

Subscription: If you would like to subscribe to our magazine, please send an email to info@haei.org or register directly at haei.org

PDF version of the magazine is available on our website – click here to see the PDF or earlier editions of Global Perspectives.

*Google Translate powers the translations on the Global Perspectives website. Google Translate is constantly improving but is not perfect. Not all text may be translated accurately, and some text may not be translated at all. HAEi is not responsible for the quality of translations. The translation should not be considered exact and is an approximation of the English language content. HAEi does not accept any liability for any damage or issues that may result from using Google Translate.

Who we are

HAE International is a global non-profit network of member organizations dedicated to raising awareness of hereditary angioedema and improving the lives of people with HAE.

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HAE International (HAEi)
10560 Main Street, Ste PS40
Fairfax City, VA 22030
United States of America
info@haei.org

Operations

HAE International (HAEi)
Vejlevej 16, 1.
8700 Horsens
Denmark
info@haei.org