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 FDA granted accelerated approval, a conditional form of approval that requires the Boston-area biotech to run a follow-up trial.
The…
[wpcode id="4722"]
[zmauto id = 1]