Medical Educational Resource
Clinical Implementation of Gene-Targeted Therapy Including Gene Replacement Therapy for Neuromuscular Diseases
This program is supported in part by an independent medical education grant from AveXis Inc.
There are a growing number of treatments that target the underlying genetic cause of neuromuscular diseases. This webinar will examine two approaches, exon skipping and gene replacement using viral vectors, being used to treat spinal muscular atrophy and Duchenne muscular dystrophy. Topics addressed include mechanism of action, technical issues, practical considerations and future directions.