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 ...

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 ...

JENKS, Okla. — A local gym is raising money to send kids with muscular dystrophy to a summer camp specialized for them. Burn Boot Camps everywhere are fundraising for the Muscular Dystrophy ...

July 26 (UPI) --A new test could speed the diagnosis of spinal muscular atrophy, a rare but deadly progressive muscle-wasting disorder, in infants. And a new way to screen for urinary tract infections ...

(CNN) — The US Food and Drug Administration has approved the first gene therapy to treat a rare and devastating muscle disease but limited the approval to kids ages 4 and 5 based on available evidence ...