Lumacaftor/Ivacaftor in Patients Aged 6–11 Years with Cystic Fibrosis and Homozygous for F508del-CFTR

Rationale: Combination lumacaftor/ivacaftor has been shown to improve lung function and other endpoints in patients aged 12 years and older with cystic fibrosis and homozygous for F508del-CFTR, but it has not been assessed in younger patients. Objectives: In this open-label phase III trial, we evalu...

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Vydáno v:Am J Respir Crit Care Med
Hlavní autoři: Milla, Carlos E., Ratjen, Felix, Marigowda, Gautham, Liu, Fang, Waltz, David, Rosenfeld, Margaret
Médium: Artigo
Jazyk:Inglês
Vydáno: American Thoracic Society 2017
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Original Articles
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5440888/
https://ncbi.nlm.nih.gov/pubmed/27805836
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1164/rccm.201608-1754OC
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