Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR

BACKGROUND: Cystic fibrosis is a life-limiting disease that is caused by defective or deficient cystic fibrosis transmembrane conductance regulator (CFTR) protein activity. Phe508del is the most common CFTR mutation. METHODS: We conducted two phase 3, randomized, double-blind, placebo-controlled stu...

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Pubblicato in:N Engl J Med
Autori principali: Wainwright, C.E., Elborn, J.S., Ramsey, B.W., Marigowda, G., Huang, X., Cipolli, M., Colombo, C., Davies, J.C., De Boeck, K., Flume, P.A., Konstan, M.W., McColley, S.A., McCoy, K., McKone, E.F., Munck, A., Ratjen, F., Rowe, S.M., Waltz, D., Boyle, M.P.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2015
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4764353/
https://ncbi.nlm.nih.gov/pubmed/25981758
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa1409547
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