Approving a Problematic Muscular Dystrophy Drug: Implications for FDA Policy

JAMA. 2016 Dec 13;316(22):2357-2358. doi: 10.1001/jama.2016.16437.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Advisory Committees / statistics & numerical data
  • Codon, Terminator / drug effects
  • Codon, Terminator / genetics
  • Drug Approval / legislation & jurisprudence*
  • Dystrophin / biosynthesis*
  • Dystrophin / genetics
  • Exons / drug effects
  • Fees, Pharmaceutical
  • Humans
  • Morpholinos / economics
  • Morpholinos / therapeutic use*
  • Muscle, Skeletal / metabolism*
  • Muscular Dystrophy, Duchenne / drug therapy*
  • Muscular Dystrophy, Duchenne / genetics
  • Muscular Dystrophy, Duchenne / metabolism
  • Randomized Controlled Trials as Topic
  • Sample Size
  • Treatment Outcome
  • United States
  • United States Food and Drug Administration*

Substances

  • Codon, Terminator
  • Dystrophin
  • Morpholinos
  • eteplirsen