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December 13, 2016

Approving a Problematic Muscular Dystrophy DrugImplications for FDA Policy

Author Affiliations
  • 1Program on Regulation, Therapeutics, and Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women’s Hospital and Harvard Medical School, Boston, Massachusetts
JAMA. 2016;316(22):2357-2358. doi:10.1001/jama.2016.16437

In September 2016, the US Food and Drug Administration (FDA) approved eteplirsen (Exondys 51), a new drug for Duchenne muscular dystrophy (DMD), overruling the recommendations of both its scientific staff and its external advisory committee. Duchenne muscular dystrophy is a progressive X-linked genetic disease caused by mutations in a gene that produces the protein dystrophin that helps stabilize muscle fibers. It is usually fatal by the third decade of life. No disease-modifying treatments are available.

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