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Efficacy and safety of ataluren in patients with nonsense-mutation cystic fibrosis not receiving chronic inhaled aminoglycosides: The international, randomized, double-blind, placebo-controlled Ataluren Confirmatory Trial in Cystic Fibrosis (ACT CF).
Konstan, M W; VanDevanter, D R; Rowe, S M; Wilschanski, M; Kerem, E; Sermet-Gaudelus, I; DiMango, E; Melotti, P; McIntosh, J; De Boeck, K.
Affiliation
  • Konstan MW; Rainbow Babies and Children's Hospital, Cleveland, OH 44106 USA; Case Western Reserve University School of Medicine, Cleveland, OH 44106 USA. Electronic address: Michael.konstan@case.edu.
  • VanDevanter DR; Case Western Reserve University School of Medicine, Cleveland, OH 44106 USA.
  • Rowe SM; Pulmonary and Critical Care Medicine, University of Alabama at Birmingham, Birmingham, AL 35249 USA.
  • Wilschanski M; Center for Cystic Fibrosis, Hadassah Hebrew University Medical Center, Jerusalem, 91240, Israel.
  • Kerem E; Center for Cystic Fibrosis, Hadassah Hebrew University Medical Center, Jerusalem, 91240, Israel.
  • Sermet-Gaudelus I; Centre de Ressource et de Competence de la Mucoviscidose, Service de Pediatrie Generale, Service de Pneumologie Pediatrique, Service de Radiologie Pediatrique, Centre d'Investigation Clinique Hôpital Necker - Enfants Malades, 75015 Paris, France.
  • DiMango E; Pulmonary, Allergy, and Critical Care Medicine, Columbia University, Department of Medicine, New York, NY 10032 USA.
  • Melotti P; Centro Fibrosi Cistica, Azienda Ospedaliera Universitaria Integrata, Verona, Italy.
  • McIntosh J; PTC Therapeutics, South Plainfield, NJ 07080 USA.
  • De Boeck K; Pediatric Pulmonology and Infectious Diseases, University Hospital of Leuven, University of Leuven, Belgium.
J Cyst Fibros ; 19(4): 595-601, 2020 07.
Article in En | MEDLINE | ID: mdl-31983658
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Full text: 1 Collection: 01-internacional Health context: 1_ASSA2030 / 6_ODS3_enfermedades_notrasmisibles Database: MEDLINE Main subject: Oxadiazoles / Drug Monitoring / Codon, Nonsense / Cystic Fibrosis Transmembrane Conductance Regulator / Cystic Fibrosis Type of study: Clinical_trials / Prognostic_studies Limits: Female / Humans / Male Language: En Journal: J Cyst Fibros Year: 2020 Document type: Article
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Full text: 1 Collection: 01-internacional Health context: 1_ASSA2030 / 6_ODS3_enfermedades_notrasmisibles Database: MEDLINE Main subject: Oxadiazoles / Drug Monitoring / Codon, Nonsense / Cystic Fibrosis Transmembrane Conductance Regulator / Cystic Fibrosis Type of study: Clinical_trials / Prognostic_studies Limits: Female / Humans / Male Language: En Journal: J Cyst Fibros Year: 2020 Document type: Article