Muscular Dystrophy Drug Repairs Gene, Helps Patients Regain Strength

October 3, 2012 | Sarepta Therapeutics announced ground breaking clinical trial results today, when their drug for Duchenne muscular dystrophy helped patients walk nearly 300 feet farther than patients on placebo, and restored a key protein lacking in some patients. Eteplirsen, the drug, repairs a gene mutation in about 13 percent of Duchenne muscular dystrophy patients, helping them produce a missing protein and regain strength, Sarepta said in its statement. Bloomberg Businessweek