The first gene therapy for children with Duchenne muscular dystrophy has been approved by the U.S. Food and Drug Administration. The therapy can be used in 4- and 5-year-olds with the degenerative ...
The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S. approval on Thursday despite concerns from some government scientists about the treatment's ability to help ...
The drug, from biotech company Sarepta Therapeutics, will need to prove in an ongoing clinical trial that it improves physical function and mobility in patients with Duchenne muscular dystrophy in ...
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