FDA approves Children's-invented gene therapy for Duchenne muscular dystrophy
The U.S. Food and Drug Administration has approved a gene therapy for Duchenne muscular dystrophy – invented at Nationwide Children’s Hospital and licensed to Sarepta Therapeutics Inc. – following months of debate over its efficacy. This is the first-ever gene therapy for Duchenne, a progressive and fatal disorder that almost exclusively affects young boys. The […]