Chest computed tomography outcomes in a randomized clinical trial in cystic fibrosis: Lessons learned from the first ataluren phase 3 study

A phase 3 randomized double blind controlled, trial in 238 people with cystic fibrosis (CF) and at least one nonsense mutation (nmCF) investigated the effect of ataluren on FEV(1). The study was of 48 weeks duration and failed to meet its primary endpoint. Unexpectedly, while FEV(1) declined, chest...

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Detalles Bibliográficos
Autores principales: Tiddens, Harm A. W. M., Andrinopoulou, Eleni-Rosalina, McIntosh, Joe, Elborn, J. Stuart, Kerem, Eitan, Bouma, Nynke, Bosch, Jochem, Kemner-van de Corput, Mariette
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2020
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7608929/
https://www.ncbi.nlm.nih.gov/pubmed/33141825
http://dx.doi.org/10.1371/journal.pone.0240898

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7608929/
https://www.ncbi.nlm.nih.gov/pubmed/33141825
http://dx.doi.org/10.1371/journal.pone.0240898

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