Efficacy and safety of lumacaftor/ivacaftor combination therapy in patients with cystic fibrosis homozygous for Phe508del CFTR by pulmonary function subgroup

BACKGROUND: Lumacaftor/ivacaftor combination therapy demonstrated clinical benefits in patients with cystic fibrosis homozygous for the Phe508del CFTR mutation. Pretreatment lung function is a confounding factor that potentially impacts the efficacy and safety of lumacaftor/ivacaftor therapy. METHOD...

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Pubblicato in:Lancet Respir Med
Autori principali: Elborn, J. Stuart, Ramsey, Bonnie W., Boyle, Michael P., Konstan, Michael W., Huang, Xiaohong, Marigowda, Gautham, Waltz, David, Wainwright, Claire E.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2016
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6612264/
https://ncbi.nlm.nih.gov/pubmed/27298017
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/S2213-2600(16)30121-7
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