Gene-targeted therapies for spinal muscular atrophy

This is a transformational time for spinal muscular atrophy management. In this video, Dr Charlotte Sumner discusses gene-targeted therapies for SMA.

Dr. Sumner has disclosed that she is/was a consultant/advisor for Ionis Pharmaceuticals, Inc., Biogen, PTC Therapeutics, AveXis, Inc., Cytokinetics, Inc., F. Hoffmann-La Roche Ltd., and Genentech, Inc.