A CFTR potentiator in patients with cystic fibrosis and the G551D mutation.

  title={A CFTR potentiator in patients with cystic fibrosis and the G551D mutation.},
  author={Bonnie W Ramsey and Jane Davies and Noel Gerard McElvaney and Elizabeth D Tullis and Scott C Bell and Pavel Drevinek and Matthias Griese and Edward F. McKone and Claire Wainwright and Michael W Konstan and Rosemari C. Moss and Felix A Ratjen and Isabelle Sermet-Gaudelus and Steven M Rowe and Qunming Dong and Sally M Rodriguez and Karl Yen and Claudia Luc{\'i}a Ord{\'o}{\~n}ez and J Stuart Elborn},
  journal={The New England journal of medicine},
  volume={365 18},
BACKGROUND Increasing the activity of defective cystic fibrosis transmembrane conductance regulator (CFTR) protein is a potential treatment for cystic fibrosis. METHODS We conducted a randomized, double-blind, placebo-controlled trial to evaluate ivacaftor (VX-770), a CFTR potentiator, in subjects 12 years of age or older with cystic fibrosis and at least one G551D-CFTR mutation. Subjects were randomly assigned to receive 150 mg of ivacaftor every 12 hours (84 subjects, of whom 83 received at… CONTINUE READING
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