GM2 PFDD Meeting

Empowering Rare Voices
to Further Drug Development

NTSAD hosted the first-ever, Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting for GM2 (Tay-Sachs and Sandhoff) with the U.S. Food and Drug Administration on February 15, 2024. The live, virtual meeting informed members of the FDA to raise their awareness about GM2. We thank all who participated, viewed, commented and supported this historic meeting for the global GM2 community.

Twenty-five families and individuals shared their experiences living with the impact of Tay-Sachs and Sandhoff diseases ranging in onsets from infantile, juvenile to late onset. Florian Eicher, MD of Massachusetts General Hospital in Boston, and Cynthia Tifft, MD, PhD of NIH in Bethesda, MD gave overviews about the diseases and the devastating effects and symptoms of these lysosomal disorders have on individuals living with them. Callers and shared comments illustrated varied experiences emphasizing the discussions at each stage of this GM2 EL-PFDD.

NTSAD represents and stands with the global GM2 Tay-Sachs and Sandhoff community as we bring the family and patient voices forward throughout this vital, critical and impactful meeting in front of the FDA, industry and those professionals dedicated to developing potential therapies for GM2.

Thank you to our sponsors