US Hereditary Angioedema Association


IN THIS ISSUE
Save the Date: 2025 US HAEA National Summit Reflect On A Milestone Year for the HAEA Community: 2024 Year in Review Inspiring the Next Generation of HAEA Leaders: Donate to the #Families4HAE Fundraiser Focus On Your HAE Health: Tips for the End of the Year	Level Up Your Skills: Apply for the HAEA Social Media Internship Program! Read the HAEA Community Blog: The Elephant in the Room: HAE & Mental Health Want to Connect with others? Join One of our Virtual HAEA CARE Groups Clinical Trial Updates Social Media Snapshots

SAVE THE DATE – 2025 US HAEA NATIONAL SUMMIT

Meet and connect with HAEA friends.
Hear about the latest advances in HAE therapies.
Be part of the movement to protect access to HAE medicines.

As always, grants are available to cover travel and accommodations.

The summit will also feature special programming of interest for:
-Youth leaders ages 12-25,
-Youngsters ages 6-12, and
-Healthcare professionals and researchers (feel free to invite anyone from your care team!)

Let's get together and embrace a brighter future for the HAEA community. We look forward to seeing you in Baltimore on July 10-13 for the 2025 US HAEA National Summit!

Registration will open on January 14, 2025, so stay tuned for more details and make sure to save the date!

Watch the 2025 Summit video HERE!

Dear HAEA friends,

As the year draws close, we gratefully reflect on a very successful 2024 resulting from the HAEA community’s truly remarkable level of participation in events and programs. We are pleased to report that 2024 was a year marked by substantial progress and innovation that pave the way for an even brighter future.

Below are some 2024 highlights:

-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 project by publishing an article documenting the development and testing of this groundbreaking tool. The publication can be viewed by clicking HERE.

-HAEA Health Team Advocates offered our community invaluable assistance in resolving insurance denials and delays, guiding and following up on referrals to expert HAE physicians, and providing answers to countless HAE-related questions.

-This year we introduced a new scholarship opportunity for Aspiring Healthcare Professionals, designed to provide financial support for those in the HAE community pursuing a career in any healthcare-related field.

Gala

-Over 250 attendees celebrated academic achievement and community spirit at the 2024 HAEA Scholarship Gala. This event reinforced the HAEA’s commitment to empowering and supporting the next generation of leaders. You can watch a recap of this event by clicking HERE.

-This year, we launched the HAEA academy, an online platform that offers a range of free online courses aimed at supporting HAEA friends at every stage of their HAE journey. You can access courses in topics like clinical trials, Medicare basics, the HAEA Shared Decision-Making Tool, and more by clicking HERE!

Youth

-This summer, the US HAEA gathered a large group of HAEA Youth Interns and Youth Leadership Council members for a two-day leadership training in Washington, D.C. These leaders-in-training learned about social media advocacy, public speaking, and how staunchly advocate for making a difference in the lives of people affected by HAE.

-The HAEA Grassroots Advocacy Network – a group of 1,021 volunteer advocates dedicated to supporting people with HAE through active participation in public policy issues – wrote letters and conducted meetings to support legislation related to a variety of issues that affect people with HAE.

-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. You can watch a recap of this event by clicking HERE.

Every member of our united HAEA community should take pride in your efforts that resulted in these noteworthy accomplishments! We wish all HAEA friends a joyful holiday season and a happy, healthy, and productive 2025!

Warm regards,

Tony Castaldo

Tony Signature

HAEA Chief Executive Officer
and Chairman of the Board

Henrik Balle Boysen

Henrik Signature

HAEA President and Board Member

Inspiring the Next Generation of HAEA Leaders: Donate to the #Families4HAE Fundraiser

Time is running out! With only a few weeks remaining in our #Families4HAE end-of-year fundraiser, we’re asking for your support to help young people affected by Hereditary Angioedema (HAE). Your generosity can make a significant difference in a young person’s life by providing the essential resources, support, and opportunities they need.

Every contribution you make goes directly towards empowering, inspiring, and educating the next generation of HAE leaders.

“Being a part of the HAEA community has played such a large role in my life. The Youth Leadership programs have allowed me to not only advocate for my younger sister, but for our community as a whole. These programs have also allowed me to make connections with many of my friends who serve as my role models, and have impacted the way I look at life.” - Ava

We are very close to reaching the fundraising goal and need your donation to create a future where young people with HAE are supported and empowered to achieve their dreams.e, support, and opportunities for a brighter future.

“The Pam King HAEA Scholarship has not only helped me believe in myself as a student, but it has helped me work towards achieving my educational and career goals by lowering the cost of my financial hardships. It has allowed me to realize that I am worthy of doing great things. This scholarship has helped me recognize that I am not alone, that there are people just like me who live with HAE every day and continue to embark on their dreams despite obstacles they may face.” - Corinne

Please take a moment to make a life-changing contribution.

Donate Now and help us ensure a brighter future for HAEA youth!

Focus On Your HAE Health: Tips for the End of the Year

The end of the year is a great time to prioritize your HAE health for the upcoming year.

Contact your physician to check to see when your prior authorization is up for renewal and if you need a visit before then.
Check in with your specialty pharmacy to update your insurance and order one last shipment before the end of the year if you are eligible for another delivery before January.
Reach out to the patient services team at your HAE medication manufacturer to check in and provide an update on your insurance plan, even if it hasn’t changed.
If you are receiving financial assistance from a charitable assistance foundation for help with your premiums or copayments, now is a great time to reach out to determine if you need to submit any additional paperwork to ensure there isn’t any delay in receiving your support.

Charitable Assistance Fund Links:
The Assistance Fund https://tafcares.org/
Accessia Health https://accessiahealth.org/

Taking these small steps before the end of the year can lessen your stress when you order your first shipment in 2025.

The HAEA Health team is here to help you with any questions you have.
Please contact us via email at health@haea.org or call us at 866-798-5598

Level Up Your Skills: Apply for the HAEA Social Media Internship Program!

Looking to grow your skills and gain hands-on experience in social media and advocacy? Are you between 17 and 25 and passionate about making a difference? We encourage you to apply for the 2025 HAEA Social Media Internship Program!

As an intern, you’ll develop valuable skills in content creation, social media strategy, and digital marketing, all while making an impact in the rare disease community. Over five months, you’ll build your resume, gain real-world experience, and contribute directly to raising awareness for HAE.

Here’s what you can expect:

Skill Development: Learn the ins and outs of social media management, content creation, and digital marketing.
Creative Opportunities: Help manage HAEA’s youth Instagram and social media channels, creating content that matters.
Community Connections: Build lasting friendships in the vibrant HAEA youth community

This internship is a perfect step if you're ready to learn, build your resume, and make a difference by informing the HAE community.

Apply to join the HAEA Social Media Internship Program and start making a difference today!

Read the HAEA Community Blog: The Elephant in the Room: HAE & Mental Health

HAE often has a significant impact on the psychological well being of people suffering from the condition as well as family members and loved ones.

It’s time to prioritize mental health within the conversation about the challenges faced by those living with HAE. Explore the emotional impact of HAE and strategies for mental wellness in this month's featured article on the HAEA Community Blog!

Read The Elephant in the Room: HAE & Mental Health by clicking HERE!
Would you like to share a unique story or opinion about your HAE? The HAEA Community Blog is the best platform to share your story with other members of the HAE community. You can share your story HERE!

HAEA CARE Groups (Community, Acceptance, Respect, Empathy)

Thank you to everyone who participated in our Self-Care Bingo night! It was a great time connecting and sharing self-care tips to help us through the end of the year.

If you weren’t about to join us, here are a few ideas that were shared that might make the end of the year less stressful:

Avoid overscheduling. With so many parties and activities this time of year it can be tempting to want to do it all. It is okay to say ‘I can’t make it”.
Focus on helping others and practicing gratitude. Giving back to others can make someone's day, inducing yours.
Go outside and get some fresh air. Even if it is cold in your area, taking a short walk outside is helpful for your body and mind.
During the busy holiday season, make sure you find some quiet time for yourself.

Wanting to connect with others? Join one of our virtual HAEA CARE Groups

We offer three times a month to connect:
1st Wednesday of the month at 7:00pm (est) / 4:00pm (pst)
2nd Thursday of the month at 11:00am (est) / 8:00am (pst)
3rd Tuesday of the month at 10:00pm (est) / 7:00pm (pst)

Interested in joining one of these monthly support groups? Contact the HAEA Health Team for more information by calling 866-798-5598 or emailing an advocate at health@haea.org.

Clinical Trial Updates

What is a Clinical Trial?
Clinical trials are research studies conducted to evaluate whether a medicine to treat HAE is safe and effective in humans. Clinical trials are a critical part of drug development and are required before a treatment can be approved by the Federal Drug Administration (FDA) and become available by prescription.

What are the benefits of participating in a Clinical Trial?
Clinical trials can offer an open-label extension, which means free access to HAE medicines being studied until they are approved and commercially available. Participation in a clinical trial also provides regular touchpoints with an HAE physician throughout the trial period and allows you to have a more active role in your health care.

Following is a list of clinical trials currently underway. If you are interested in receiving more information on clinical trials, or are interested in participating in any of the trials listed below, please reach out to the HAEA Research Team at research@haea.org.

The RAPIDe-3 Trial

The US HAEA is currently assisting Pharvaris in recruiting people for the RAPIDe-3 trial, a Phase 3 worldwide clinical research study to investigate the safety and effectiveness of an investigational oral drug (deucrictibant) to relieve the symptoms of Hereditary Angioedema (HAE) attacks. This is a placebo-controlled study, and if you choose to participate you will take capsules to treat a total of two HAE attacks. The use of an approved on-demand medication to treat acute attacks is permitted and will be provided at no cost.

You may qualify for the RAPIDe-3 trial if you are diagnosed with HAE Type I or II and be between the ages of 12 and 75 years old, with at least two attacks in the past three months and have had experience using standard-of-care treatment to manage on-demand treatment for HAE attacks. After completing the RAPIDe-3 Study, participants may be eligible to join an open-label extension (OLE) study, and continue receiving deucrictibant to treat acute HAE attacks at no cost.

The HAELO Study

The US HAEA is currently assisting Intellia in recruiting people with HAE for the HAELO study.

The HAELO study is a phase 3 study to evaluate the efficacy and safety of NTLA-2002 compared to placebo in adults with HAE. This investigational drug uses CRISPR gene editing to potentially treat genetic diseases.

To qualify for the HAELO study, participants must be 18 years of age or older with a clinical history consistent with HAE Type 1 or Type 2. This study will help researchers learn if the study drug is safe and effective for people with HAE when compared with placebo.

The OASIS-HAE Plus Study

The US HAEA is currently assisting IONIS in recruiting people with HAE for the OASIS-HAE Plus study.

The OASIS-HAE Plus study is an open-label extension study without placebo, evaluating the safety and effectiveness of an investigational subcutaneous preventive medicine called donidalorsen, which is given once every 4 or 8 weeks. This investigational drug uses a technology called antisense, which targets the kallikrein generating system using messenger RNA to suppress the protein that produces kallikrein.

To qualify for the OASIS-HAE Plus study, participants must be 12 years of age or older with a documented diagnosis of HAE. This study will help researchers learn if the study drug is safe and effective for people with HAE when given over a long-term period. Participants in the OASIS-HAE Plus study are either switching over from a different prophylaxis therapy, or continuing on from another donidalorsen study.

CSL312_3003 Pediatric Study

The US HAEA is currently assisting CSL Behring in recruiting children ages 2-11 with HAE for the CSL312_3003 study, which will investigate the safety and efficacy of subcutaneous CSL312 for prophylactic treatment of pediatric-age individuals with HAE.

KONFIDENT-KID Study

The US HAEA is currently assisting Kalvista in recruiting children ages 2-11 with HAE for the KONFIDENT-KID study, a worldwide clinical research study to investigate the efficacy of an investigational oral drug (sebetralstat) to relieve the symptoms of Hereditary Angioedema (HAE) attacks in adolescent patients.

If you have HAE Type I, Type II, or HAE with Normal C1-Inhibitor and are interested in learning about new clinical studies to prevent attacks, please contact an HAE advocate at 866-798-5598 to see if there will be an active clinical trial site in your area.

Social Media Snapshots

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Update your HAEA membership information HERE!


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The HAEA is an advocacy and research organization committed to actively engaging our community in a wide variety of grassroots activities that promote HAE education and awareness. We provide personalized services to address the unique needs of people with HAE and their families, which includes helping them secure access to and reimbursement for modern HAE medicines. Our great success in supporting clinical research has resulted in a variety of FDA-approved therapeutic options. We work closely with expert physicians to continuously upgrade our community’s quality of life through improving diagnosis and knowledge of the condition, and encourage an individualized approach to selecting an optimal treatment. The HAEA is product and company neutral and continues to enthusiastically support drug discovery research aimed at the next generation of HAE therapies. Copyright © (2024) US Hereditary Angioedema Association All rights reserved. No part of this publication may be reproduced, stored in a retrieval system, or transmitted in any form by any means, mechanical, electronic, photocopying, recording, or otherwise without the prior written permission of the US Hereditary Angioedema Association. For information, send an email to info@haea.org.