FDA greenlights #Sarepta Therapeutics’ milestone treatment for #Duchenne muscular dystrophy – MedCity NewsMedCity News

Originally posted 2016-09-20 16:46:55.


The FDA’s approval of the drug, Exondys 51, also known generically as eteplirsen, came over the objections of its own advisory committee, which voted not to approve the medication earlier this year. Patients and their families had lobbied hard for the drug, made by Sarepta Therapeutics of Cambridge, Massachusetts, noting that people with the disease have few treatment options.

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