Research Opening Session

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

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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