Ivacaftor in a G551D homozygote with cystic fibrosis

N Engl J Med. 2013 Sep 26;369(13):1280-2. doi: 10.1056/NEJMc1213681.
No abstract available

Publication types

  • Case Reports
  • Letter

MeSH terms

  • Adolescent
  • Aminophenols / therapeutic use*
  • Chlorides / analysis
  • Cystic Fibrosis / drug therapy*
  • Cystic Fibrosis / genetics
  • Cystic Fibrosis Transmembrane Conductance Regulator / genetics*
  • Female
  • Forced Expiratory Volume / drug effects
  • Homozygote
  • Humans
  • Mutation
  • Quinolones / therapeutic use*
  • Sweat / chemistry

Substances

  • Aminophenols
  • Chlorides
  • Quinolones
  • Cystic Fibrosis Transmembrane Conductance Regulator
  • ivacaftor