Young Boy Becomes First DMD Patient to Receive Investigational Systemic Microdystrophin Gene Therapy
The first Duchenne muscular dystrophy (DMD) patient has received systemic microdystrophin gene therapy as part of a Phase 1/2 clinical trial at Nationwide Children’s Hospital in Columbus, Ohio. The trial (NCT03375164), currently recruiting participants, will test the safety and efficacy of a single dose of intravenous microdystrophin gene therapy…