December 21, 2021
On October 28, 2021, the APBD Research Foundation engaged the U.S. Food and Drug Administration (FDA) in a Patient-Led Listening Session to share the burden of APBD and the needs of our community.
Our Foundation applied for and received approval from the FDA to host the APBD Listening Session with the FDA.
During the Listening Session, representatives from our Foundation and the broader community shared their personal APBD experiences as well as its impact on their family members. They also discussed the diagnostic challenges and the physical, emotional, and financial toll that are, unfortunately, echoed in so many patients’ experiences. Given that there are no treatments or a cure for APBD, they thoughtfully expressed what would be meaningful impacts when it comes to a potential therapy.
Jeff Levenson, co-president of the APBD Research Foundation, shared, “We illuminated for FDA staffers the lived experience of APBD and how it impacts and destroys lives. We also expressed our commitment to bring the patients’ perspective to every stage of therapeutic development and approval.”
This meeting was only the beginning of an ongoing conversation with the FDA. We look forward to sharing more updates as we move this important work forward.
News Releases
The APBD Research Foundation is preparing to host a Vision Loss and APBD Focus Group to convene later this fall. This group will bring together APBD patients who have experienced or are experiencing changes to their vision, caregivers, and clinicians in a 1.5 hour session to learn about lived experiences with vision-related symptoms and begin to understand how vision loss is associated with APBD.
The APBD Research Foundation is excited to announce a new collaboration with Costello Medical, Inc., following the Foundation’s successful application to Costello Medical’s pro bono project initiative for Rare Disease organizations.
We are honored to share the story of an extraordinary act of generosity and planning by former Board member David Epstein. David passed away on January 3, 2026.
The APBD Research Foundation is delighted to announce that Lindsay Gill, PhD has been promoted to full-time Research Director.
We are thrilled to announce that our community raised an incredible $201,177 during the 2026 APBD Tour de Friends Rally for Research fundraising campaign, exceeding our original goal of $120,000.
The Academy of General Dentistry (AGD) recently awarded the APBD Research Foundation's co-president Dr. Jeff Levenson with the Lifelong Learning & Service Recognition (LLSR). He received this award along with 36 other LLSR recipients during the AGD’s scientific session in Las Vegas, NV on June 27, 2026.
In April 2026, the APBD Research Foundation announced participant recruitment for its APBDRF Drug Repurposing Study initiative in partnership with COMBINEDBrain. We are grateful to the patients and families in our community that have volunteered to provide biosamples to help make this study a reality.
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.