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

  • Eitan Kerem
  • , Michael W. Konstan
  • , Kris De Boeck
  • , Frank J. Accurso
  • , Isabelle Sermet-Gaudelus
  • , Michael Wilschanski
  • , J. Stuart Elborn
  • , Paola Melotti
  • , Inez Bronsveld
  • , Isabelle Fajac
  • , Anne Malfroot
  • , Daniel B. Rosenbluth
  • , Patricia A. Walker
  • , Susanna A. McColley
  • , Christiane Knoop
  • , Serena Quattrucci
  • , Ernst Rietschel
  • , Pamela L. Zeitlin
  • , Jay Barth
  • , Gary L. Elfring
    • Ellen M. Welch, Arthur Branstrom, Robert J. Spiegel, Stuart W. Peltz, Temitayo Ajayi, Steven M. Rowe

Research output: Contribution to journal › Article › peer-review

307 Scopus citations

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Keyphrases

Medicine and Dentistry

Biochemistry, Genetics and Molecular Biology

Pharmacology, Toxicology and Pharmaceutical Science