Eteplirsen Approved for Duchenne Muscular Dystrophy: The FDA Faces a Difficult Choice

Mol Ther. 2016 Nov;24(11):1884-1885. doi: 10.1038/mt.2016.188.
No abstract available

Publication types

  • Editorial

MeSH terms

  • Drug Approval*
  • Humans
  • Male
  • Morpholinos / therapeutic use*
  • Muscular Dystrophy, Duchenne / drug therapy*
  • Oligonucleotides
  • United States
  • United States Food and Drug Administration

Substances

  • Morpholinos
  • Oligonucleotides
  • eteplirsen