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

Canavan

GM1 Gangliosidosis

GM2 Gangliosidosis (Infantile/Juvenile)

Late Onset GM2

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

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