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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Detalhes bibliográficos
Publicado no:PLoS One
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: Public Library of Science 2020
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7608929/
https://ncbi.nlm.nih.gov/pubmed/33141825
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0240898
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