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
