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.
Kesselheim AS, Avorn J. Approving a Problematic Muscular Dystrophy DrugImplications for FDA Policy. JAMA. 2016;316(22):2357–2358. doi:10.1001/jama.2016.16437
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