The APBD Research Foundation is pleased to announce the third of our 2025 Rally for Research Pilot Grant Program grant recipients: Rebecca Koch, PhD, RDN at Duke University. Her research project is entitled, Gene Therapy to Treat the Neurophenotype of Glycogen Storage Disease Type IV.

The APBD Research Foundation is pleased to announce the second of our 2025 Rally for Research Pilot Grant Program grant recipients: Mayank Verma, MD, PhD at UTSW Medical Center. His research project is entitled Preclinical Validation and Biomarkers in APBD of a GYS1-ASO Currently in Phase 1 Clinical Trial for Lafora Disease

On behalf of the APBD community, the APBD Research Foundation has responded to the US Food and Drug Administration's (FDA) request for public comments for its recently released draft guidance for the use of the Plausible Mechanism Framework for individualized genetic therapies. 

We are excited to share that our global genetic prevalence of GBE1-related diseases study has been published in the Journal of Inherited Metabolic Disease Reports (JIMD Reports).

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.