Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Duchenne Muscular Dystrophy

This nonrandomized controlled trial analyzes safety, biological, and functional outcomes associated with the infusion of rAAVrh74.MHCK7.micro-dystrophin gene transfer in a small group of patients with Duchenne muscular dystrophy.
Source: JAMA Neurology - Category: Neurology Source Type: research