Ivacaftor for patients with cystic fibrosis

  title={Ivacaftor for patients with cystic fibrosis},
  author={C. Wainwright},
  journal={Expert Review of Respiratory Medicine},
  pages={533 - 538}
  • C. Wainwright
  • Published 2014
  • Medicine
  • Expert Review of Respiratory Medicine
  • Ivacaftor is an oral bioavailable potentiator of the cystic fibrosis transmembrane conductance regulator protein. It is the first therapeutic agent that has been registered for clinical use which targets the basic defect in people with cystic fibrosis who carry a G551D mutation or other rarer specific gating mutations. Clinical trials have shown consistent and impressive clinical benefit that appears to be sustained over time in people with cystic fibrosis who carry a G551D mutation and similar… CONTINUE READING
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