Leading the worldwide fight to treat and cure
Tay-Sachs, Canavan, GM1 and Sandhoff diseases

NTSAD Virtual Family Conference Research

Research Opening Session

Frances Platt, PhD, Oxford University
"Therapies for Lysosomal Disease: Progress and Challenges" (for the four diseases NTSAD supports)

Opening Research Presentations
Moderated by Jamie Ring, NTSAD Board member

Speakers: Gerald Cox, MD, PhD, Clinical Trials Overview, Patroula Smpokou, MD, FDA's Role in Drug Development, Rachel Witten, MD, Gene Therapy and the FDA

Research Breakouts


GM1 Gangliosidosis

GM2 Gangliosidosis (Infantile/Juvenile)

Late Onset GM2

Email here with any questions you may have regarding these breakouts.