From Mariam Movsisyan, HAE Armenia

A Milestone for Hereditary Angioedema Care in Armenia: First Successful Pregnancy in a Patient with HAE Type III.

Armenia has reached an important milestone in the management of hereditary angioedema (HAE) with the successful pregnancy and delivery of a healthy baby by a woman diagnosed with HAE type III—the first such documented case in the country.

The patient, a young woman with recurrent episodes of angioedema without C1-inhibitor deficiency, was diagnosed with HAE type III prior to pregnancy. Given the known challenges associated with hormonal influences on disease activity, pregnancy was initially considered high risk. However, following pre-pregnancy counseling and close multidisciplinary supervision, a carefully individualized management plan was developed.

Throughout pregnancy, the patient was regularly monitored by allergists-immunologists and obstetricians experienced in high-risk care. Emphasis was placed on prophylaxis, early recognition of symptoms, and preparedness for acute attacks. Importantly, treatment decisions were guided by international HAE recommendations and adapted to local clinical practice.

Despite the physiological and hormonal changes of pregnancy, the patient experienced no life-threatening attacks. Delivery was completed without complications, and both mother and newborn remained healthy in the postpartum period.

This case represents a significant achievement for HAE care in Armenia, demonstrating that with timely diagnosis, expert counseling, and coordinated care, women with HAE type III can safely plan pregnancy and achieve favorable outcomes. It also highlights the growing awareness and capacity for managing rare diseases in the region.

The Armenian HAE community hopes this experience will encourage earlier diagnosis, patient education, and confidence among women with HAE who wish to build families.

From Ahtashamul Habib Ovi, HAE Bangladesh

HAE Bangladesh has taken a significant step forward in advancing awareness and understanding of Hereditary Angioedema (HAE) by completing the translation of essential patient education materials into Bengali. These materials, including the HAE emergency card, are now available on haei.org and are accessible to all patients seeking information on HAE, including its diagnosis, management, and treatment.

Through our newly launched website hosted by HAEi, we are successfully connecting an increasing number of patients across the country, and the national HAE patient community continues to grow. To further support patient access to care, the website now includes a curated list of hospitals and specialist physicians, enabling patients to connect with appropriate healthcare providers more easily.

In preparation for hae day :-) 2026, HAE Bangladesh is planning a full-day awareness event to be held at a renowned hospital in Bangladesh. This initiative aims to strengthen national awareness of HAE and formally raise the HAE flag as a symbol of advocacy and solidarity.

Currently, C1 esterase inhibitor injections are not commercially available in Bangladesh. To address this critical gap, we have established a pharmaceutical channel with neighboring India, enabling patients to order the medication and receive delivery in Bangladesh. At the same time, we are actively working toward making this treatment directly available within Bangladesh in the near future.

From Mdm Zhang Nan, HAE China, and Lim Yong Hao, RPA HAEi

Hosting an Impactful Annual HAE Conference
In 2025, we hosted the 9th Annual HAE Patient Conference in China, bringing together more than 200 patients, family members, and healthcare professionals, making it the largest patient-focused HAE conference to date. Alongside the conference, a large-scale free clinic was organized at Peking Union Medical College Hospital, where over 70 patients received consultations. Close to 20 patients underwent initial screening, creating important opportunities for earlier diagnosis and timely intervention.

Supporting Drug Development and Expanding Treatment Options
Over the past year, three new HAE therapies entered clinical trials in China. Through patient engagement and information-sharing, we supported more than 100 patients in making informed, voluntary decisions to participate in these trials. Their involvement has been vital in advancing research, accelerating clinical development, and contributing to the future availability of new treatment options for HAE patients in China.

Growing the Patient Community and Extending Support
Our patient community continued to grow steadily in 2025. Through outreach and media engagement, membership increased by more than 90 individuals, bringing our total membership to 420. We remain committed to building a supportive and inclusive community, ensuring that every patient has access to reliable information, peer support, and a strong sense of belonging.

Patient Advocacy Priorities for 2026
Looking ahead to 2026, our advocacy efforts will focus on two key priorities:

1. Identifying Undiagnosed Patients
   We will strengthen public awareness initiatives and deepen engagement with primary healthcare institutions to improve recognition of HAE. We aim to shorten the diagnostic journey for undiagnosed patients and connect them earlier to appropriate medical care and support networks.
2. Improving Access to Innovative Treatments
   We will continue to support patient participation in clinical trials and work with stakeholders to improve pathways for access to innovative HAE therapies. Our goal is to ensure that patients in China can benefit from timely, equitable access to a broader range of treatment options and greater autonomy in managing their condition.

From Costa Rica

Throughout 2025, patients with Hereditary Angioedema in Costa Rica have been working together to advance awareness of the disease. In our country, Law 10.550, PREVENTION AND COMPREHENSIVE HEALTH CARE FOR PEOPLE WITH RARE DISEASES, TO IMPROVE THEIR QUALITY OF LIFE AND THAT OF THEIR FAMILIES, was recently passed, and Hereditary Angioedema is included in this law. This has been a significant step forward because, like other rare diseases, we could receive more support and care in the future.

As part of our efforts, we have held events with pharmaceutical representatives to learn more about the medications available in Costa Rica and their effectiveness for patients. We have also held meetings with other important patient organizations in the country, such as ANASOVI and the World Rare Disease Day Network Organizing Committee, to validate the inclusion of Hereditary Angioedema (HAE) in Law 10.550. They provided guidance on government procedures and how the new law will function, and both organizations offered their support and advice on any future issues.

Additionally, we successfully lobbied for the issuance of ID cards so that people with HAE can present them during emergencies at hospitals and health centers throughout the country. We have also held fundraising events, raising the necessary funds to finally legally establish the Costa Rican Angioedema Association.

To better focus on our short- and long-term goals, we are holding bi-weekly meetings for the first time with patients and the Board of Directors created by the patients, as well as meetings with the representative of the Hereditary Angioedema International Patients’ Organization (HAEi) for Central America and the Caribbean. We have also established communication plans with legal representatives to consolidate the Costa Rican Patients Association, and communication with medical specialists in HAE in Costa Rica.

From Rosa María Pérez González, HAE Cuba

We, the Cuban patients with Hereditary Angioedema, send you our warmest greetings and express our respect and gratitude for the work carried out by the International Society (HAEi) on behalf of those of us living with this disease worldwide. For our community, your work represents a source of hope, support, and constant companionship.

In the current context of Cuba, marked by a sustained economic, social, and health crisis, it is necessary to share with you that access to specialized diagnostic tools and modern medications indicated for the treatment of Hereditary Angioedema remains impossible. Currently, specific therapies are unavailable in the country, and in the face of acute, life-threatening crises, the only available treatment option is fresh frozen plasma.

Nevertheless, the Cuban medical community continues its efforts to conduct clinical studies on individuals with compatible symptoms or a family history of the condition, with the aim of identifying them and providing them with medical follow-up, with a view to future opportunities for diagnosis and treatment. Both patients and healthcare professionals remain open and receptive to any possibility of international aid and cooperation that will allow us to advance the study and treatment of Hereditary Angioedema in Cuba.

From HAE Dominican Republic

The year 2025 represented a period of strategic and sustained work for the hereditary angioedema patient community in the Dominican Republic. Although many of the actions undertaken during this period did not receive immediate public attention, steady progress was made in fundamental processes aimed at generating real and lasting impacts for patients, their families, and caregivers.

Throughout the year, collaborative work was carried out to build a solid foundation that will allow, in the medium term, the inclusion of medication for the treatment of hereditary angioedema within the national healthcare system. This objective has guided institutional efforts, as well as the actions taken at the technical and medical levels.

During the last quarter of 2025, significant progress was made by reaching out to and establishing relationships with professionals from various healthcare fields, including immunology, intensive care, surgery, dentistry, and anesthesiology. These specialists have expressed interest and willingness to actively collaborate in strengthening comprehensive patient care and achieving the established objectives. This medical support network is expected to continue expanding in the coming period.

The year ends with a positive outlook and clear expectations for 2026, which is projected to be a year of consolidation, growth, and significant progress for the patient community, the allied medical team, the foundation, as well as for the families and caregivers who accompany them throughout this process. The institution remains firmly committed to finding solutions that guarantee a better quality of life and timely access to appropriate treatments for those living with hereditary angioedema in the country.

From Raquel Fuentes, HAE El Salvador

During 2025 in El Salvador, awareness of Hereditary Angioedema (HAE) has been actively promoted through several initiatives, such as the distribution of informational flyers in community health clinics, with the aim of raising awareness among both patients and healthcare personnel about this rare condition. In addition, meetings were organized to coordinate a walk with patients to commemorate HAE Day in the country, creating a space for gathering, support, and visibility that fosters education, empathy, and unity within the community around HAE. Meetings were also held with physicians to identify more patients in the country, and efforts were made to establish connections with government representatives.”

From Samantha Alvarado, HAE Guatemala

Guatemala does not yet have a fully implemented law on rare diseases, but in late 2024, several members of Congress introduced Initiative 6451, which seeks to create the Law for Comprehensive Care for Patients with Rare, Difficult-to-Diagnose, or Genetically Based Diseases. This effort is currently underway, having been referred to the Health and Social Assistance Committee for its review and corresponding report; however, its approval may take longer. As a group of patients with HAE, we have been working to schedule meetings with key individuals to help advance the initiative.

In Guatemala, as in some other Latin American countries, the main challenges are late diagnosis and the availability of specific therapies for HAE. Furthermore, approximately 44% of the population is indigenous, primarily belonging to Mayan, Garifuna, and Afro-descendant communities. Therefore, limited access to health services is often concentrated in urban areas, leaving indigenous communities with little coverage. Cultural and linguistic barriers exist: many Indigenous women and men face discrimination in hospitals and difficulties communicating in their own language. Hospitals typically use general therapies: corticosteroids and antihistamines, which are ineffective for HAE, are often used initially due to a lack of knowledge. Therefore, throughout 2025, we have been working together to continue raising awareness about Hereditary Angioedema in the country.

From Makiko Matsuyama, HAE Japan

HAEJ (HAE Japan) has remained highly active throughout the year, implementing initiatives almost every month. One of our key priorities has been to clearly communicate the role and value of patient organizations—not only to the general public, but also to physicians—by reaffirming the importance of patient associations within the medical community.

Public awareness activities were a major focus in 2025. At hae day :-) in May, we once again conducted the nationwide Purple Light-Up campaign following last year’s success. A total of 48 landmarks and towers across Japan were illuminated in purple, including large-scale sites such as Tokyo’s Rainbow Bridge, generating significant media coverage. In February, HAEJ organized Rare Disease Day (RDD) Kobe, an event of high interest to pharmaceutical companies, and cooperated on an interview article published by a major national online media outlet. In addition, Japan hosted the World Expo this year, and on May 23 (Intractable Diseases Day), we were invited to speak about HAE at an RDD event held at the Expo. Members of the Imperial Family were also in attendance, and the event received wide media coverage, becoming a valuable opportunity for awareness.

Patient engagement continued to expand nationwide. Patient networking meetings were held across Japan, including—for the first time—one in Kagoshima. Many patients still face difficulties accessing appropriate treatment, particularly in regional areas, and we believe it is essential to reach beyond major cities. This year, the HAEJ website was also fully renewed with support from pharmaceutical partners, resulting in a modern and well-designed platform. We will continue to strengthen website operations and regularly update resources for patients and families.

Engagement with the medical community remained strong. HAEJ exhibited booths and delivered presentations at dermatology, otolaryngology, and allergy society meetings, including a session at the Dermatological Society meeting that received an exceptional standing ovation from attending physicians. We also actively participated in media seminars and public lectures organized by pharmaceutical companies. Looking ahead, we value continued collaboration through international meetings such as the APAC Conference and the upcoming Madrid conference, and we hope for more opportunities to engage with colleagues from Europe and the United States. With new treatments expected to launch in 2026, the treatment environment in Japan continues to improve, and we look forward to further expanding our activities with energy and commitment.

From Patricia Karani, HAE Kenya

Giving a Voice to Patients Living with Hereditary Angioedema in Kenya

For many people in Kenya living with Hereditary Angioedema (HAE), the journey to diagnosis and appropriate care remains long, confusing, and often isolating. Between October and December 2025, HAE Kenya intensified its advocacy efforts to ensure that patients are not only seen within the healthcare system but heard, understood, and supported.

A significant step toward patient empowerment during this period was the completion of the Understanding HAE booklet in the Swahili language. As Kenya’s national language, Swahili plays a critical role in reaching out to a larger audience of patients and families, especially in areas where Swahili is more conversant than English. By making HAE education accessible in a familiar language, HAE Kenya is helping patients better understand their condition, advocate for themselves in clinical settings, and engage more confidently in their care—particularly in rural and underserved communities.

Recognizing that awareness must be matched with access, HAE Kenya also updated its website to serve as a practical resource for patients seeking care. The platform now includes up-to-date information and a growing list of healthcare professionals trained in HAE diagnosis and management across the country. For patients living far from the ACARE Centre based in Nairobi, this has been a critical development, offering reassurance that knowledgeable medical support can be found closer to home and reducing the burden of long-distance travel for already vulnerable families.

Advocacy efforts extended directly into healthcare facilities through a collaborative awareness initiative with Dr. Priya Bowry of the ACARE Centre at the Coast General Teaching and Referral Hospital in Mombasa. The session brought together doctors, nurses, medical students, and pathologists, creating a vital space for dialogue between clinicians and the patient community. Central to this engagement was the sharing of a patient journey to diagnosis—an experience that illuminated the real consequences of delayed recognition, misdiagnosis, and limited treatment options. By centering the patient voice, the discussion challenged healthcare providers to consider not only the clinical presentation of HAE but its profound impact on quality of life, mental well-being, and family dynamics.

Healthcare professionals were encouraged to maintain a high index of suspicion for HAE and to view early diagnosis as a form of patient advocacy in itself. The visibility of HAE Emergency Department posters displayed within the hospital further reinforced this message, serving as a constant reminder that rare diseases, though often overlooked, require readiness and informed response.

Beyond awareness, HAE Kenya continued to support newly identified patients and their families by facilitating appropriate referrals through the ACARE Centre. For many, this marked a turning point—ending years of uncertainty and opening a pathway to effective treatment and informed care. The gratitude expressed by families underscored a simple but powerful truth: access to the right care can transform lives.

These efforts reflect HAE Kenya’s broader advocacy mission—to build a healthcare environment where rare disease patients are not marginalized, where language and geography do not determine outcomes, and where patient voices help shape systems of care. While challenges remain, the progress made during this period affirms that sustained advocacy, partnership, and patient leadership are essential to improving the lives of those living with Hereditary Angioedema in Kenya.

From HAE Panama

2025 has been a year of very positive results for patients in Panama. After several years of meetings with the Panamanian Ministry of Health and the Office of Rare Diseases, the official treatment guidelines for patients with HAE were finally approved and distributed to all hospitals and health centers in the country. This has opened new doors, and we hope to continue making progress in the treatment of HAE in Panama.

As patients and members of the Association, we have always worked together toward the same goals. The most important has always been access to the appropriate medication for HAE. We have already obtained approval for some medications and continue working to secure approval for others that are more advanced. In addition, more patients have been diagnosed.

As a group, we will continue working to ensure that patients learn to manage the disease and that more is known about HAE.

From HAE Puerto Rico

The Puerto Rico community proudly participated in the 2025 US HAEA Summit in Baltimore, where members connected with fellow HAEA advocates and gained valuable insights into the latest HAE news from knowledgeable healthcare professionals.

In November 2025, more than 70 members of the Puerto Rico community came together in San Juan, PR, for the 2025 Community Connections Lunch. The event provided a meaningful opportunity to share personal stories, exchange contact information, welcome new members, and strengthen community bonds.

Additionally, several members of the Puerto Rico community were invited to attend the 2026 Global Angioedema Leadership Conference, which will take place in Madrid, Spain, in March 2026—highlighting the community’s growing leadership and engagement on a global level.

From Alicia Campos, HAE Spain (Google Translated from Spanish)

A year of change and new goals for our association
This year has marked a turning point for our association. For the first time, we have moved our annual meeting to Vall d’Hebron Hospital in Barcelona. This change inaugurates a new model of rotating meetings among the main CSURs (Centers, Services, and Reference Units) for angioedema in Spain, with the aim of strengthening collaboration with healthcare professionals and facilitating the participation of patients and families from all regions.

Our goal for 2026 is to reach a greater number of patients, ensure broader and more equitable access to modern treatments, and contribute to improving the quality of life of all those affected. We will continue working to ensure that the real needs of the community are heard, recognized, and addressed, promoting policies and strategies that generate a positive and lasting impact.

We are thrilled to announce that we are already preparing our annual meeting, which next year will be held for the first time at the Virgen del Rocío Hospital in Seville on February 21, 2026, continuing the rotating venue model we began this year.

In addition, we are organizing another edition of the Camino de Santiago pilgrimage, scheduled for March 19-24. This unique experience combines camaraderie, visibility, and personal growth, and has become one of the most highly valued activities among our members.

From Marijk Beekman-Kortekaas, President, HAE the Netherlands

Reflecting on 2025 and Looking Ahead: HAE the Netherlands
As we close the year 2025, I feel proud to look back on what we have achieved together as HAE the Netherlands. This year has been filled with milestones that strengthened our community and given us new energy to move forward.

Patient Information Day and Members Day
One of the highlights of the year was our Patient Information Day. This event has become a cornerstone of our work, offering patients and families the chance to learn about the latest medical developments, treatments, and research. But beyond the science, it is also a day where people can share their personal stories, ask questions, and feel the support of others who truly understand what it means to live with HAE.

Earlier in the year, we hosted our Members’ Day, which was equally special. This gathering is always a moment of celebration, where we reflect on what we have accomplished as an association and discuss the challenges that lie ahead. It is a day filled with energy, laughter, and solidarity. For many of our members, these events are not just informative; they are a lifeline, a reminder that they are part of a larger family.

The Success of Noa and the Youngsters’ Community
Another milestone in 2025 was the success of Noa, who completed the LEAP program. Her project, creating a youngsters’ community for HAE patients, is now well underway. This initiative is incredibly important. Young people living with HAE often face unique challenges: navigating school, friendships, relationships, and future plans while managing a rare disease. Having a dedicated community where they can connect, share experiences, and support each other is vital.

Noa’s work shows us the power of youth leadership. She is building something that will last, something that ensures the next generation of HAE patients has a voice and a platform. Watching her progress has been inspiring, and it gives me confidence that the future of our association will be shaped by young people who are passionate, creative, and determined.

Our Role in the National Alliance for Rare Diseases
On a national level, HAE the Netherlands continues to play a leading role. We hold the most important seat on the board of the Dutch National Alliance for Rare Diseases, which gives us a strong position to advocate for patients not only with HAE but across the spectrum of rare diseases.

Through this alliance, we are actively involved in drafting the new National Plan for Rare Diseases, which will be launched in 2026. This plan is a crucial step forward. It will set the agenda for how rare diseases are recognized, treated, and supported in the Netherlands. Being part of this process means that the voices of patients are heard at the highest level. It ensures that our experiences, our struggles, and our strengths are reflected in national policy.

The Power of the Global HAE Community
What makes these achievements even more meaningful is that they are part of something bigger: the global HAE community. Across countries and continents, we are united by shared experiences and a common mission. We learn from each other, we inspire each other, and we support each other.

The progress we make in the Netherlands is connected to the progress being made elsewhere. When one country develops new initiatives, others can follow. When one patient shares their story, it resonates across borders. Together, we are stronger than the challenges we face. Together, we can achieve more than any of us could alone.

This sense of global solidarity is what gives me hope. It shows that HAE is not just a rare disease; it is a worldwide movement of patients, families, doctors, and advocates working side by side. And it proves that when we join forces, we can change lives and shape the future.

Looking Ahead to 2026
As we look forward to 2026, our hopes are clear. We want to continue building on the successes of 2025. That means once again organizing both a Patient Information Day and a Members’ Day, ensuring that our community has opportunities to come together, learn, and celebrate.

But we also want to take an exciting new step: for the very first time, we plan to host a special event dedicated to young people living with HAE. This event will be designed specifically for them, giving them a platform to share their experiences, ask questions, and build connections. We believe this will be an important milestone in our work, one that strengthens the youth community and ensures that younger members feel seen and supported.

Conclusion
2025 has shown us what we can achieve when we work together. It has been a year of connection, empowerment, and progress. As we move into 2026, we are determined to build on these successes, to strengthen our community, and to ensure that every voice, young and old, is part of the future of HAE the Netherlands.

Together with the global HAE community, we will continue to make HAE visible, to advocate for better care and support, and to create a world where everyone living with HAE feels heard, valued, and empowered.

From Jess Myers,  Communications and Advocacy Manager, US HAEA

Key US HAEA Highlights in 2025

1,400 US HAEA Friends Share a Memorable 2025 HAEA National Summit in Baltimore, Maryland

The 2025 HAEA National Summit, held in Baltimore, Maryland, was an incredible success! From July 10–13, more than 1,400 HAEA community members, including people with HAE, their caregivers, medical professionals, researchers, and industry sponsors, came together for an unforgettable weekend of education, empowerment, and connection.

We look forward to continuing this momentum and building on the connections made in Baltimore as we work toward a future where everyone with HAE has access to the medicine and support services they deserve.

US HAEA Publishes First-Ever Study Showing the Number of People Diagnosed with HAE in the United States
Recently published in a top-rated peer-reviewed medical journal, this landmark HAEA-initiated study featured collaboration with leading HAE experts and specialists in data mining and demographic analysis.

We now estimate that nearly 10,000 people in the U.S. have received a diagnosis of HAE. As noted earlier, this number includes all forms of the condition—not just those caused by C1-INH deficiency—and provides the clearest picture yet of the HAE population in the U.S.

US Congress Acknowledges the Need for Research on HAE with Normal C1 Inhibitor
In a Senate appropriations committee report released in June, legislators stated, “The Committee applauds the previous work across National Institutes of Health (NIH) to identify genetic mutations that cause HAE and to advance treatment options and care for affected individuals, moving a once deadly rare disease to a largely manageable chronic illness. However, the Committee notes that challenges persist in diagnosis, treatment, and care for HAE patients with Normal C1 Inhibitor and encourages the NIH to work with other Institutes and Centers, and the stakeholder community, to advance research in this key area.”

The tireless efforts of the HAEA community have resulted in elected officials finally understanding the unmet needs of those with HAE with Normal C1 Inhibitor. We are excited to see this progress in an official congressional record, and we look forward to working with legislators to improve diagnosis, treatment, and access.

Looking Towards A Positive Future for US HAEA Members in 2026
In 2026, the US HAEA hopes that people living with HAE experience greater confidence, independence, and peace of mind in their daily lives. We envision a future where timely diagnosis is the norm, treatment options are accessible, and no one faces HAE alone. Through continued advocacy, education, and research, we strive for a world in which people with HAE can fully pursue their goals, knowing their condition is understood, well-managed, and supported at every stage of life.

Create a True Partnership with your Physician to Better Manage Your HAE
HAEA Friends have access to an exciting (and free!) resource to prepare for their next HAE appointment: The HAEA Shared Decision Making Tool (HAEA SDM).

By answering a series of questions, HAEA SDM summarizes:

• Recent experiences with HAE,
• Current treatments, and
• Preferences and what matters most when considering an HAE treatment.

This information is compiled into a downloadable one-page summary overview that serves as a basis for a more collaborative treatment-oriented discussion with your HAE physician.

HAEA SDM will help our community and their doctors create a treatment plan that truly aligns with individual needs.

The HAEA SDM Tool is available here: https://www.haea.org/pages/p/sdm_tool

HAEA Community Blog
The HAEA Community Blog is a platform that allows people with HAE to share their unique stories on a wide variety of topics, ranging from emotional health, navigating insurance challenges, and the journey to diagnosis and treatment.

Featured Blog Articles

“Traveling with Hereditary Angioedema (HAE) may require extra planning, but it doesn’t have to hold you back. Whether it’s a road trip, international adventure, or quick weekend getaway, being prepared can give you the confidence to enjoy your journey safely.”

6 Tips to Prepare for Traveling with HAE at https://www.haea.org/pages/bp/Blog35_Travel

“If I could tell you to wake up tomorrow and do one thing, it’s to use your voice. No matter how small your follower count is, no matter how unimportant you think your story is, every single voice and story matters.”

Read Gen Z Advocacy in 2025 at https://www.haea.org/pages/bp/blog36_Zach

Research Updates
The HAEA’s research activities focus on generating data and publishing studies that advance the understanding of HAE’s impact on the lives of our community. Our 2026 research program is outlined below:

• HAEA Quality of Life Instrument Short Form: In 2024, we published a medical journal article introducing a new HAE Quality of Life Instrument (HAE-C1INH-QoL). This HAE-specific tool addresses the limitations of currently available options and better captures our community’s experience with HAE. We recently completed our work to develop and validate a short-form version. We have prepared a manuscript that will be submitted to a medical journal in early 2026.
• Study Validating Our HAE-CIINH-QOL Instrument for Patients Diagnosed with HAE Normal C1-Inhibitor: Our HAE-C1INH-QoL instruments (long and short form) are only validated in people with HAE due to C1 inhibitor deficiency. It is important that members of our community diagnosed with HAE with normal C1INH also have tools to measure their quality of life. We have completed a study that validates the HAE-C1INH-QoL instruments for the HAE normal C1INH population using two datasets that included 160 participants. We are preparing a manuscript, which will be submitted to a medical journal in mid-2026.
• Pediatric Study on HAE: We are in the process of completing data-gathering using an innovative, validated approach where children express the burden of HAE through artwork they create. This research is being designed to produce high-quality data that highlights unmet needs and issues that are important to the pediatric population. We expect this project to result in a peer-reviewed medical journal publication that we will submit later in the second quarter of 2026.
• The Economic and Socio-economic Burden of HAE Caregivers: The HAE burden of illness and its wide-ranging economic and socio-economic impact on individual patients are well known and detailed in an HAEA study published in 2021. What is less well understood, however, is how the quality of life and well-being of caregivers are affected by looking after people with HAE. Moreover, to our knowledge, there is little, if any, data that quantifies caregiver burden. We designed and conducted a comprehensive survey that characterizes the caregiver experience. We are in the process of analyzing the data and preparing a medical journal manuscript that will be submitted in the summer of 2026.