Editor's Note: This oil pastel artwork was created by patient Alma Hecht. Our thanks to the Hecht family for sharing it with our community.



APBD Researchers Share Backstories!

We interviewed Matthew Gentry, PhD and Berge Minassian, MD -- two Million Dollar Bike Ride research grant recipients -- to give you a chance to get to know them and read about their groundbreaking APBD research. Read more by clicking on the links below:

Dr. Gentry and Dr. Minassian, thank you for your vision and your efforts. We wish you and your team much success on behalf of our community!

Golden Heart Flower, Ltd. Granted FDA Orphan Drug Designation for GHF-201 for the treatment of Glycogen Storage Diseases Type IV, including APBD

Golden Heart Flower, Ltd. announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation for the company’s product GHF-201 to treat glycogen storage disease type IV, including APBD.



Golden Heart Flower (GHF) – an Israel-based pharmaceutical company founded by a family with a member diagnosed with APBD – is focused on developing therapies for APBD and other rare glycogen storage and lysosomal storage diseases. GHF-201 is the company’s lead small molecule, discovered following a long study by researchers Dr. Or Kakhlon from Hadassah Ein Kerem Medical Center (Israel) and Professor Miguel Weil from Tel Aviv University (Israel). GHF-201 is capable of degrading polyglucosan bodies, the culprit in APBD. In an APBD mouse model study, GHF-201 was effective in lowering polyglucosan bodies, improving mobility, and extending lifespan.

In Memory of Those We Lost in 2023

It is with heavy hearts that we acknowledge the individuals in our community who passed away this year. We honor their beauty and their strength. Our thoughts are with their family members and friends.

MISSED IT? Watch Tracy Boggs, PT, MPT, NCS, talk about Fall Risk, Recovery Techniques, and Prevention Considerations

You can also watch recordings of previous Chats here.

Upcoming Events

From Chat events to workshops and scientific symposiums, the Foundation hosts programs for patients, caregivers, and health professionals which connect our community members and deepen the understanding of how APBD impacts lives.

These events offer peer support, opportunities to hear from health experts and Foundation leaders, and the chance to raise funds for APBD research.

Patient Chats

Jan. 24, 2024, 8pm ET | 5pm PT

Feb. 28, 2024, 8pm ET | 5pm PT

The Patient Chat is hosted by volunteer moderator Harriet Saxe. To learn more, email Harriet at harriet@apbdrf.org.

Caregiver / Family Chats

Jan.11, 2024, 7:30pm ET | 4:30pm PT  

Feb. 8, 2024, 7:30pm ET | 4:30pm PT

This Chat is hosted by volunteer moderator Linda Cedarbaum. To learn more, email Linda at linda@apbdrf.org.

Help Us Finish 2023 Strong!

We’re aiming to raise $120,000 by the end of the year to pave the way for greater APBD breakthroughs in 2024. We ask that you keep the APBD Research Foundation in mind as you make your year-end giving decisions.

Your contributions sustain and make life-changing work possible, including: 

• advancing cutting-edge research
• advocating for APBD patients’ needs
• offering peer-to-peer support

Thanks to 83 individual donors, we have raised $97,423 to date.



Will you help us end the year strong by contributing to our $120,000 goal? 

Every gift sustains the Foundation’s work and changes lives.

Make a Transformational Gift Through Your IRA

If you are 70½ years old or older, you can make a tax-free gift to the APBD Research Foundation directly from your individual retirement account (IRA). This is called a qualified charitable distribution (QCD) and was authorized by the Secure Act 2.0. The new law took effect January 1, 2023. Make a transformational gift today and save on taxes when you support the APBD Research Foundation.

We provide this FREE monthly newsletter resource to all of our community members.

This program is made possible by contributions to the APBD Research Foundation from donors like you.

Thank You!