FDA approves Sarepta’s controversial drug for Duchenne muscular dystrophy

(By Ed Silverman for STAT)

he Food and Drug Administration on Monday approved1 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. Continue reading article here……

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