APBD Research Foundation 2025 Rally for Research Grant Series: UTSW Medical Center Scientist Awarded Pilot Grant to Evaluate Treatment for an Allied Disease in APBD
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
Foundation Submits Public Comment to the FDA on the Plausible Mechanism Framework for Accelerating Individualized Therapy Development for Rare Diseases
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has responded to the US Food and Drug Administration's (FDA) request for public comments for its recently released draft guidance for the use of the Plausible Mechanism Framework for individualized genetic therapies.
Advancing APBD Research Has Never Been More Urgent or More Promising
We are excited to launch the 2026 APBD Tour de Friends Rally for Research Fundraising Campaign to raise $150,000.
We Need You for the APBDRF Drug Repurposing Study!
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.
Carol Rosenstock Joins the APBD Research Foundation’s Board of Directors
The APBD Research Foundation is delighted to announce the appointment of Carol Rosenstock to its Board of Directors.
Developing a "Disease Concept Model" for APBD -- a Collaborative Research Initiative
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.
APBD Research Foundation 2025 Rally for Research Grant Series: UCLA Scientists Awarded Pilot Grant to Discover New APBD Biomarkers
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.
n-Lorem Foundation ASO for the Deep Intronic Mutation Causing APBD Receives FDA Approval for n-of-1 Clinical Trials
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.
We’re Expanding Our APBDRF BioBank
The APBD Research Foundation is excited to announce its 2026 goal of recruiting 30 participants to its GSD IV BioBank!
APBD Research Foundation Mourns the Passing of Former Board Member and Director Emeritus David Epstein
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.