Organization: Sarepta
Gene Therapy Approaches to Treatment We covered the rationale behind gene therapy approaches for Duchenne and received updates from Pfizer, Sarepta Therapeutics, REGENXBIO Inc., and Solid BioSciences.
Senior Director of Clinical Science Solid Biosciences
A moderated panel discussion focusing on the potential challenges and complications of AAV therapy and the clinical strategies to make dosing AAV more successful.
Professor and Associate Chair
University of Florida, Department of Pediatrics
Chief, Neuromuscular and Neurogenetic Disorders of Childhood Section National Institute of Neurological Disorders and Stroke/NIH
Director, Center for Gene Therapy Nationwide Children's Hospital
Co-Founder and Chief Scientific Officer
Kate Therapeutics