US Hereditary Angioedema Association


IN THIS ISSUE
Heart Health Month Rare Disease Day 2022 Legislative Update Pam King HAEA Scholarship HAEA Round Table Physician Spotlight	Community Blog HAE Speaks Podcast #BeyondHAE Youth Podcast Clinical Trial Updates Education Webinar Virtual Meet & Greets

February is Heart Health Month

Taking care of your heart isn't just about eating healthy and exercising. It is also important to focus on your mental health which can improve your overall physical heart health. Studies show that 80 percent of cardiovascular diseases may be preventable with education and action. Simple lifestyle changes can make a big impact when it comes to heart health! Peace of mind and lots of laughing can help your heart stay young and healthy! It is important to care for your emotional state as much as you care for your physical well being. Seek activities that nurture your spirit, encourage positivity and build strong bonds with friends and family who bring joy to your daily life.

Self Care is an act of LOVE

• Schedule time for yourself
• It's okay to say NO
• Talk to a friend
• Step outside and get fresh air
• Spend time with pets - they help lower your blood pressure
• Unplug from social media and take time out from the news
• Take deep slow breaths and feel your lungs expand
• Listen to music

Consider joining a Hereditary Angioedema (HAE) Virtual Support Group! Connecting with others, sharing our stories and struggles, and laughing together are just one of the ways these groups help our community members. You can join an HAEA Virtual Support Group by reaching out to troyce@haea.org.

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.

Join us on February 28th to celebrate Rare Disease Day!

This February 28th, join the over 300 million people who live with a rare disease throughout the world to raise awareness about the challenges presented by rare diseases and to advocate for better healthcare, access to diagnosis and effective and affordable therapies.

Since its creation in 2008, Rarediseaseday.org has provided a worldwide platform that advances rare disease advocacy work at the local, national and global landscapes.

We invite you to raise HAE awareness in your community throughout the month of February.

• Follow us on Social Media and share our content to family and friends.
• Share the recently published US HAEA Medical Advisory Board Treatment Guidelines for Managing Hereditary Angioedema with all of your healthcare providers. Download here.
• Download or order a copy of the HAEA Insurance Reimbursement Guidebooks for HAE therapies here.
• Order and deliver free HAE ER Toolkits to urgent care centers, emergency rooms, school nurses, and first responders – to order an HAE ER Toolkit click here.
• Request the HAEA Children's Books be donated to a local school library, click here for more information.
• Donate your HAE attack swelling pictures in support of the HAEA's education programs by clicking here.
• Create your own awareness event – click here to learn more.

For more information about HAE visit www.haea.org!

HAEA Webinar: 2022 Legislative Update

2022 is shaping up to be one of the most important years 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 cordially invite you to participate in a webinar on Thursday, March 3 at 7:00 PM ET, during which we will discuss how you can ensure our voices are heard in Washington and anywhere else where there are challenges that could hinder access to and reimbursement for HAE therapies.

The webinar will cover

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

Speakers:
Anthony Castaldo (US HAEA President & CEO)
Dane Christiansen (HMCW)
Jim Romano (United for Charitable Assistance)

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.

"The HAEA Scholarship has lightened the financial burden of college and allowed me to attend the school of my dreams. The HAEA Scholarship program has also provided opportunities for me to get more involved in HAEA and meet other college students and children with Hereditary Angioedema." ~ Sydney Peel

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 first event in the HAEA Round Table series will be on Tuesday February 22nd at 6:30 PM ET. This event will feature children ages 5-11 and will showcase their answers to questions related to their personal experiences in living with HAE.

HAEA Round Table Calendar:

• February 22nd
For Kids with HAE Ages 5-11 - How Does It Feel to Be a Kid with HAE?
• 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?

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 US HAEA Medical Advisory Board physician, Raffi Tachdjian, MD.

"I discovered a more expanding world in allergy and immunology in medical school, then it clicked. I found the right balance of treating adults and children with allergic conditions, while being a detective in solving complex immunological conditions that puzzle us. Research has always been a part of my studies and career. I feel like we need to do more as physicians in discovering the deepest details of conditions we treat, develop optimal therapies, and always remember to treat the patient as a whole person."

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

HAEA Community Blog

Do You Have A Story to Share About Your HAE?

The HAEA is excited to announce the creation of the HAEA Community Blog, a space for individuals to share their experiences in their own words with the HAEA community!

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

Stay tuned for the official launch of the HAEA Community Blog!

The HAEA is looking for individuals interested in sharing their own story through the HAEA Community Blog! Share your story here.

Contact 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 else you listen to your podcasts!

Episode 22: In the February episode of the HAE Speaks podcast, Bobbi shares her story of living with HAE and describes how the evolution of treatments has vastly improved over the years.

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 - HAE and Dating

Youth Podcast series

Listen to the #BeyondHAE Youth Podcast, a space for young HAE community members to share their stories with the world!

In this episode of the #BeyondHAE podcast, Kelsie, a young individual with HAE, discusses how she approached introducing her diagnosis to her partner, Matt, who joins in as a guest host. She discusses the ways that he supports her in her diagnosis and he talks about what it means to him to support her.

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

HAEA Treatment Education Series Webinar

Two Journeys, One Goal: Raise Expectations of Living with a Rare Disease

Date: Wednesday February 16th, 2022

Time: 7:00 PM E.T. / 4 PM P.T.

Speakers:
Liz (HAE Patient Ambassador)
Chaeli (HAE Patient Ambassador)
Cheryl Schwartz (SVP US Rare Disease Business Unit at Takeda)
Renee Siegel (Patient Education Marketer at Takeda)

Sponsored by: Takeda

Webinar Link: https://haea.zoom.us/j/81785611943
Phone Number: +1 646 558 8656
(Webinar ID: 817 8561 1943)

HAEA Virtual Meet & Greet

Are You Interested In Meeting Others In The HAE Community?

Do you have questions for others living with HAE? Have you been recently diagnosed with HAE? Are you a person living with HAE or a caregiver? Are you interested in sharing your own HAE journey with other members of the HAE community?

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! 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?

"Have a notebook ready so you can write down helpful information throughout the presentation."
~ Jami A.

Upcoming Haea Virtual Meet & Greet Events

Tuesday, February 15th, 6:30 PM P.T.

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