European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene

Neuromuscul Disord. 2015 Jan;25(1):5-13. doi: 10.1016/j.nmd.2014.11.011. Epub 2014 Nov 24.
No abstract available

MeSH terms

  • Adolescent
  • Adult
  • Child
  • Child, Preschool
  • Clinical Trials, Phase II as Topic
  • Codon, Nonsense*
  • Drug Approval*
  • Dystrophin / genetics*
  • Humans
  • Male
  • Muscular Dystrophy, Duchenne / drug therapy*
  • Muscular Dystrophy, Duchenne / genetics*
  • Oxadiazoles / therapeutic use*
  • Randomized Controlled Trials as Topic
  • Treatment Outcome
  • Young Adult

Substances

  • Codon, Nonsense
  • Dystrophin
  • Oxadiazoles
  • ataluren