US Hereditary Angioedema Association


IN THIS ISSUE
hae day :-) Protect the Rights of People with HAE! Pam King HAEA Scholarship HAEA Round Table Health Team Update Physician Spotlight	Community Blog HAE Speaks Podcast #BeyondHAE Youth Podcast Clinical Trial Updates Education Webinar Virtual Meet & Greets

Amplify Rare Stories On hae day :-) 2022

May 16th is hae day :-), an internationally recognized Day of Hereditary Angioedema Awareness. The HAEA kicked off our upcoming celebration of hae day :-) on Rare Disease Day with an awareness campaign.

Each of you have compelling stories about what it's like to live with HAE. Your life experiences can teach others about the HAE community's challenges and opportunities to achieve greater health and quality of life. We are asking you to share your personal stories to elevate and amplify our voices for HAE health, and we are providing you with free storytelling tools to do it!

Whether you prefer to present your unique story in written or video formats, we can't wait to hear from you.

We want to flood social media with our stories on May 16th, as we commemorate the 10th anniversary of hae day :-)

When you pledge your participation, we will send you everything you need to create a memorable story to share with your personal networks on hae day :-) 2022. The storytelling toolkit we will send to you includes: a tripod, access to our storyvine recording system, and a thorough training on how to tell your story!

Share Your Rare Story Here!

Protect the Rights of People with HAE!

2022 is shaping up to be a critical year for HAEA grassroots legislative advocacy. Our Advocacy Team anticipates changes to national healthcare policies that are likely to have a significant impact on our community.

We would like to thank everyone who joined us for the 2022 HAEA Welcome to Congress and Legislative Update, but if you missed it, you can still participate by (1) watching the recording of the live event, and (2) engaging in direct outreach with your congressional representatives to ask for specific HAE-related protections and action.

If you would like additional information on how to get in touch with your local legislator, or are interested in participating in the HAEA's volunteer grassroots program, please contact jessica@haea.org.

The webinar covered

• Congress's new priorities
• Issues that will impact the HAE community in 2022
• What you can do to defend access to and reimbursement for HAE therapies
• How you can protect financial assistance programs that help with the cost of HAE medicines

Watch the live recording of the event here!

HAEA Updates

The Pam King HAEA Scholarship Program is NOW Accepting Applications for the Fall 2022 Semester

Fall 2022 Scholarship Applications are now open!

Are you planning to attend a college or university in the Fall of 2022? The HAEA helps students with a confirmed HAE diagnosis pursue their academic goals by reducing the financial burden associated with higher education. Applications for the Pam King HAEA Scholarship Program are open twice a year. We invite interested individuals to take advantage of this opportunity, and complete your application before March 31st.

"Having support from the HAEA community means so much to me, and I am extremely thankful for receiving this scholarship. Because of it, I can stress less about the expenses of attending college and focus more on my studies and giving back to the community." ~ Alexis

Fill out the Scholarship Application HERE!

HAEA Round Table

Join us at the HAEA Round Table, where members of our community will be able to talk about how they have dealt with the unique challenges of HAE. The HAEA Round Table events will premiere on Facebook Live, so make sure to tune in to the US HAEA official Facebook page to learn more about how others manage their HAE!

The next event in the HAEA Round Table series will be on Tuesday, April 12th at 6:30 PM ET. This event will feature children ages 12-15 and will showcase their answers to questions related to their personal experiences managing Anxiety, Stress, and Growth with HAE.

HAEA Round Table Calendar:

• April 12th
For Kids with HAE Ages 12-15 - Anxiety, Stress, and Growth with HAE
• June 14th
For Youth with HAE Ages 16-20 - How Do You Talk to Others About Your HAE?
• August 9th
For Young Adults with HAE - Growing Into Adulthood with HAE
• October 11th
For Baby Boomers with HAE - How Does HAE Affect Aging?
• November 1st
For Caregivers and Parents - What is the Best Way to Support Someone with HAE?

If you missed the February HAEA Round Table for Kids ages 5-11, you can access the recording here!!

Health Team Update

March is Eye Wellness Month!

While we all continue to be hopeful that we can get back to living a normal life, many of us are still living "virtually" and spending a lot of time in front of a computer screen. Whether it be for work, video chatting with family and friends, perusing Facebook, or online shopping, we must be conscientious of the negative effects of increased digital screen time on our eyes.

Symptoms of eye strain include tired, burning or itching eyes, dry eyes, blurred vision, and headaches. The American Optometric Association recommends these tips to help protect our vision:

• Keep screen 20-26 inches away from your eyes and slightly below eye level
• Adjust your lighting to reduce glare and harsh reflections
• Use computer glasses with yellow tinted and anti-reflective lenses that block blue light and help ease eye strain
• Take frequent breaks using the 20-20-20 Rule (Every 20 minutes look away from your screen and look at an object 20 feet away for 20 seconds). This helps the eyes reset and replenish themselves

Please remember to incorporate these tips into your daily routine. We need to take care of our eyes today to help maintain healthy vision.

The HAEA Health Team is always here for you! Please reach out to us at 866-798-5598 for assistance with 1.) insurance issues, 2.) finding an HAE expert physician, 3.) updating your member information, 4.) joining a virtual support group, or 5.) an HAE-related question.

Throughout 2022, the US HAEA will spotlight Hereditary Angioedema (HAE) treating physicians who have made an impact within the HAE community.

This month, we will be recognizing HAE treating physician, Aleena Banerji, MD.

"The most rewarding part of my job has been seeing the improvement in quality of life for all my the people with Hereditary Angioedema (HAE). I love hearing stories about my patients taking vacations, participating in activities that they were not able to do previously, and at times almost “forgetting” they have HAE. This is a tough disease, but seeing these improvements and hearing these stories always brings a smile to my face and makes me happy I went into the medical field!"

Thank you Dr. Banerji for your dedicated service to the HAE community!

HAEA Community Blog

The HAEA is excited to announce that we have officially launched a Community Blog! A space where you can share your experiences in your own words with the HAEA community!

This blog highlights stories written by HAE community members, for HAE community members. A space to amplify our voices through our experiences, reflections, perspectives, and more!

Featured Story:

The Caregiver Journey: Team Jack: Ally, caregiver to her brother, Jack, describes the unique way that her family advocates for HAE in her town's annual St. Patrick's Day parade.

"Raised in a large and very Irish family, Saint Patrick's Day has always been one of the most important holidays of the year for us. In 2019, we decided that Team Jack needed its own section to march within our town's Union County Irish American parade, so our mom reached out to the US HAEA for help. With their aid, our family connections, and some serious building skills from my mom’s husband, Team Jack was in the parade! We marched with every friend and family member who could come from all over. HAE families, friends, and HAEA Staff from New York, Ohio, Florida, and all over New Jersey were able to join us! We all donated our Team Jack shirts and walked the parade route through my hometown. We even had our own float decked out in HAEA banners and signs!"

You can read more of Ally's story now on the HAEA Community Blog here.

Interested in sharing your own story through the HAEA Community Blog? Click here.

Contact HAE Advocate, Hannah Carroll at hannah@haea.org for more information!

Podcasts

HAE Speaks Podcast

Download and listen to HAE Speaks, a podcast where people with HAE get the chance to broadcast their stories to the world! HAE Speaks gives you the opportunity to learn more about your own HAE through the shared experiences presented by our monthly hosts. You can download HAE Speaks on Apple Podcasts, Spotify, or wherever you listen to your podcasts!

Episode 23: Troyce, an HAEA Health team member, provides information and tips about insurance in 2022.

LISTEN NOW

Do you have a topic, question, or idea that you would like us to cover? Send your suggestion to Mike Mallory at mikemallory@haea.org.

#BeyondHAE Youth Podcast - Learning To Feel Comfortable With My Condition

Youth Podcast series

Episode 36 is hosted by Zanya, a young individual with HAE, who talks about her diagnosis journey and some of the challenges she has encountered along the way. She talks about how getting involved in HAE advocacy and awareness has helped her come out of her skin and feel more comfortable sharing her diagnosis with people in her life.

#BeyondHAE is the only podcast around made exclusively by young people with HAE. Listen to hear different perspectives of how you can live a normal life with HAE. #BeyondHAE is available to download on Apple Podcasts, Spotify, or wherever you get your latest podcasts!

LISTEN NOW

Clinical Trial Updates

What is a Clinical Trial?

Clinical trials are research studies conducted with people affected by HAE to evaluate whether a drug is safe and effective in humans. They are one of the final stages of the medical research and development process and follow strict standards to protect the health and safety of participants. 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 access to HAE medicines at no cost to the participant for the duration of the trial, before it is available to everyone. 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. Your participation in a trial is your decision, and you have the ability to withdraw if the trial is not working for you, without judgment or jeopardizing 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.

Normal C1-Inhibitor Trial

The US HAEA continues to support Takeda’s efforts in recruiting people with HAE with Normal C1-Inhibitor for their clinical trials. Individual sites will comply with state and local guidelines and safety protocols for bringing individuals into clinics. There are currently 14 active sites in the US.

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.

CSL312_3002 Study

The US HAEA is currently assisting CSL Behring in recruiting people with HAE for the CSL312_3002 Garadacimab study, a worldwide clinical research study to investigate the safety and effectiveness of an investigational subcutaneous medication given once a month to prevent Hereditary Angioedema (HAE) attacks. In this study, doctors will be evaluating the long term use of garadacimab.

The trial is currently fully recruited for adult participants, however, a few more adolescent participants (ages 12 - 17) are needed to meet their required numbers.

This is an open label study with no placebo phase required, which means that all participants will receive garadacimab for at least one year, and the use of an on-demand medication to help with acute attacks is permitted.

RAPIDe-1 & CHAPTER-1 Studies

The US HAEA is currently assisting Pharvaris in recruiting people with HAE for two clinical research studies to investigate the safety and effectiveness of an investigational oral drug for both acute and prophylactic use of Hereditary Angioedema (HAE) attacks.

In the RAPIDe-1 study, doctors will be evaluating how effective three different doses of the study medication (PHVS416) are in relieving symptoms associated with HAE attacks. The study drug is in the form of soft capsules taken by mouth. In the CHAPTER-1 study, doctors will be evaluating the effectiveness of PHVS416 as a potential oral treatment to prevent HAE attacks.

To qualify for the RAPIDe-1 study, participants must be diagnosed with HAE Type I or II and be between the ages of 18 and 75 years old, with at least 3 attacks in the last four months or 2 attacks in the last two months.

To qualify for the CHAPTER-1 study, participants must be diagnosed with HAE Type I or II and be between the ages of 18 and 75 years old, with at least 3 attacks in the last 3 months or 2 qualifying attacks during the screening period. The use of an approved on-demand medication to treat acute attacks is permitted.

The OASIS Study

The US HAEA is currently assisting IONIS in recruiting people with HAE for The OASIS study, which aims to find out whether the study drug (called donidalorsen) is safe and effective at reducing or preventing HAE attacks.

This is a Phase 3 worldwide placebo-controlled trial where doctors are studying donidalorsen injections to determine safety and effectiveness at reducing or preventing HAE attacks. Participants may be eligible to participate in an open-label extension study at the end of The OASIS study, where all participants receive donidalorsen.

To qualify for The OASIS study, participants must be 12 years of age or older with a documented diagnosis of HAE. Participants must experience at least two attacks during the screening period, and have access to and the ability to use at least one or more acute medications to treat HAE attacks.

KOMPLETE Study

The US HAEA is currently assisting Kalvista in recruiting people with HAE for the KOMPLETE study to understand the effects of an investigational oral medication called KVD824 in adults 18 years of age and older with HAE type I or type II.

This is a placebo-controlled trial where doctors are studying three different doses of KVD 824-201 to see which one may be best as a safe prophylaxis treatment for HAE attacks.

To qualify for the KVD 824-201 study, participants must be 18 years of age or older and diagnosed with HAE Type I or II. You will also need to have access to an on-demand medication to use for HAE attacks, and be willing to use a study-approved method of birth control during the study.

HAERMONY1 Study

The US HAEA is currently assisting BioMarin in recruiting people with HAE for the HAERMONY1 study, which is an investigational gene therapy called BMN 331 to improve HAE symptoms in adults 18 years of age and older diagnosed with HAE due to C1-INH deficiency.

To qualify for the HAERMONY1 study, participants must be 18 years of age or older and diagnosed with HAE due to C1-INH deficiency. You will also need to use preventative medication to prevent attack or an on-demand medication during HAE attacks. Participants must also have at least one HAE attack on average per month. *Additional study requirements apply.

For those who qualify, participation lasts about 5 years after receiving the study drug. There are weekly and monthly visits during the first year. After the first year, the visits occur every three or every six months.

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.

HAEA Treatment Education Series Webinar

A Shared Voice: Developing a Hereditary Angioedema Treatment Plan with Your Physician

Date: Wednesday, March 30th, 2022
Time: 7:00 PM E.T. / 4 PM P.T.
Speaker: Marc Riedl, MD, MS
Sponsored by: BioCryst
Webinar Link: https://haea.zoom.us/j/83609962301
Phone Number: +1 646 558 8656
(Webinar ID: 836 0996 2301)

HAEA Virtual Meet & Greet

Are You Interested In Meeting Others In The HAE Community?

Do you have questions for others living with HAE? Or would you just like to network with other HAEA members?

The HAEA Virtual Meet & Greet Events are a perfect way to reach out and get to know others affected by HAE. Each month HAEA staff will be presenting on programs, resources, and relevant HAE topics as well as providing an opportunity for the HAE community to meet each other. If you are interested in participating in an HAEA Virtual Meet & Greet, please check out the HAEA Events Calendar, or reach out to Mike Mallory at mikemallory@haea.org.

What would you tell someone who is considering attending an HAEA Meet & Greet?

"A HAEA Meet & Greet helps you feel like you are not alone. It was great to meet with others who have this disease and to hear about so many avenues of help. A definite good use of time. So glad I attended!"
~ Ruth R.

Upcoming Haea Virtual Meet & Greet Events

Tuesday, March 15th, 6:30 M.T.

Tuesday, April 19th, 6:30 PM P.T.


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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 © (2022) 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.