Be a light of hope this holiday season. Ignite more progress, restore more freedom, and inspire more hope for families living with neuromuscular disease.

Medical Educational Resource

We're accelerating the delivery of treatments and cures.

Clinical Implementation of Gene-Targeted Therapy Including Gene Replacement Therapy for Neuromuscular Diseases

Activity Snapshot

Activity Type: 
On-Demand Webinar
Release Date: 
Friday, October 25, 2019
Sponsor Information: 

This program is supported in part by an independent medical education grant from AveXis Inc.

Faculty

Diana Bharucha-Goebel, MD
Diana Bharucha-Goebel, MD
Pediatric Neuromuscular Neurologist, Assistant Professor

Children's National Hospital, National Institute of Health/ NINDS

Clinical Implementation of Gene-Targeted Therapy Including Gene Replacement Therapy for Neuromuscular Diseases
On-Demand
Activity Overview: 

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.