Fda Approves First Drug For Duchenne Muscular Dystrophy
The Food and Drug Administration on Monday approved a controversial drug to treat Duchenne muscular dystrophy, a rare disease that confines boys to wheelchairs and condemn them to an early death. The decision came after months of protracted debate about whether drug maker Sarepta Therapeutics had provided enough evidence to demonstrate that its medication, called eteplirsen, had a meaningful impact on patients. In reaching its decision, the agency essentially overruled its own medical staffers, who earlier this year questioned the effectiveness of the drug over concerns about a small clinical trial....