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

Author:

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

Publisher

Elsevier BV

Subject

Pulmonary and Respiratory Medicine

Reference34 articles.

1. A new era in the treatment of cystic fibrosis: correction of the underlying CFTR defect;Boyle;Lancet Respir Med,2013

2. Association of a nonsense mutation (W1282X), the most common mutation in the Ashkenazi Jewish cystic fibrosis patients in Israel, with presentation of severe disease;Shoshani;Am J Hum Genet,1992

3. Correlation between genotype and phenotype in patients with cystic fibrosis;N Engl J Med,1993

4. Genotype-phenotype correlations in cystic fibrosis;Kerem;Pediatr Pulmonol,1996

5. Genotype-phenotype correlation for pulmonary function in cystic fibrosis;de Gracia;Thorax,2005

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