Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.

@article{Kerem2014AtalurenFT,
  title={Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.},
  author={Eitan Kerem and Michael W Konstan and Kris de Boeck and Frank J. Accurso and Isabelle Sermet-Gaudelus and Michael Wilschanski and J Stuart Elborn and Paul Melotti and Inez Bronsveld and Isabelle Fajac and Anne Malfroot and Daniel B. Rosenbluth and Patricia Ann Walker and Susanna A McColley and Christiane Knoop and Serena Quattrucci and Ernst Friedrich August Rietschel and Pamela L Zeitlin and Jay Barth and Gary L. Elfring and Ellen Marie Welch and Arthur A. Branstrom and Robert J. Spiegel and Stuart W. Peltz and Temitayo Ajayi and Steven M Rowe},
  journal={The Lancet. Respiratory medicine},
  year={2014},
  volume={2 7},
  pages={
          539-47
        }
}
BACKGROUND Ataluren was developed to restore functional protein production in genetic disorders caused by nonsense mutations, which are the cause of cystic fibrosis in 10% of patients. This trial was designed to assess the efficacy and safety of ataluren in patients with nonsense-mutation cystic fibrosis. METHODS This randomised, double-blind, placebo-controlled, phase 3 study enrolled patients from 36 sites in 11 countries in North America and Europe. Eligible patients with nonsense-mutation… CONTINUE READING
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