Title: Gene Therapies for Spinal Muscular Atrophy and Duchenne Muscular Dystrophy: Translating Benchside Promise into Clinical Practice
Speaker(s):
Emma Ciafaloni, MD, FAAN
Professor of Neurology, Pediatrics, and Ob&Gyn
Department of Neurology
University of Rochester
Learning Objectives:
1. Understand the current treatment landscape for SMA and DMD.
2. Understand the new natural history of DMD and SMA.
3. Understand the risks and benefits of in vivo gene therapy for muscle and anterior horn diseases in children.