We are excited to launch the 2026 APBD Tour de Friends Rally for Research Fundraising Campaign to raise $150,000.

The APBD Research Foundation is excited to announce an opportunity for you to play a direct role in advancing treatment research. You do not have to leave home to participate.

The APBD Research Foundation is delighted to announce the appointment of Carol Rosenstock to its Board of Directors.

The APBD Research Foundation is excited to partner with COMBINEDBrain on the development of a “Disease Concept Model” for APBD and other neuromuscular forms of GSD IV.

The APBD Research Foundation is pleased to announce the first of our 2025 Rally for Research Pilot Grant Program grant recipients: Gal Bitan, PhD and Brent Fogel, MD, PhD.

The APBD Research Foundation is excited to share with our community that the n-Lorem Foundation has received FDA approval to proceed with n-of-1 clinical trials for their treatment targeting the APBD deep intronic mutation.

The APBD Research Foundation is excited to announce its 2026 goal of recruiting 30 participants to its GSD IV BioBank! 

New York, NY – The APBD Research Foundation shares with sadness the passing of former Board Member and Director Emeritus David Epstein on January 3, 2026.

The estimated global genetic prevalence of GBE1-related diseases — APBD and early-onset forms of Glycogen Storage Disease Type IV included — has been updated from 1 in 325,000 (reported in 2022) to 1/236,000. This updated estimate suggests that up to 34,000 people worldwide could be affected. 

The APBD Research Foundation is excited to share that Matthew Gentry, PhD -- a member of our Scientific and Medical Advisory Board -- -- has been selected as 1 of 10 recipients of the Oxford-Harrington Rare Disease Centre’s 2025 Rare Disease Scholar Awards.

The APBD Research Foundation is excited to announce the launch of the GSD IV BioBank in partnership with COMBINEDBrain.

As the APBD Research Foundation and our research community embark on new and continued research studies to find cures for and advance our understanding of APBD, we strongly encourage our patient community to create a Clinical Research ID (CRID).

Nano-rare disease advocates, clinicians, researchers, and industry collaborators gathered in Cambridge (MA) earlier this month for the n-Lorem Foundation’s 2025 Nano-rare Patient Colloquium. 

Over 900 rare disease advocates, clinicians, researchers, industry partners, and other leaders gathered earlier this month in Washington, DC for the National Organization for Rare Disease’s (NORD) 2025 Breakthrough Summit.

The National Organization for Rare Disorders (NORD) is launching the Living Rare Study, and we need you and everyone in the GSD IV community to participate.

Carol Rosenstock recently opened up about her journey from being diagnosed with MS to receiving an APBD diagnosis in the Jewish Standard.

There are many challenges for small, scattered rare disease communities like ours. To address some of those challenges, the APBD Research Foundation recently became a member of the Consortium for Outcome Measures and Biomarkers for Neurodevelopmental Disorders (COMBINEDBrain).

New York, NY – The APBD Research Foundation shares with deep regret the passing of our co-founder Michael Weiss on September 6, 2025 from the consequences of APBD.

We’re excited to share that Golden Heart Flower, Ltd. (GHF) has just launched a Phase I clinical trial in Europe to study GHF-201 in adult healthy volunteers.