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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 […]
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