Efficacy and safety of the elexacaftor/tezacaftor/ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial

BACKGROUND: Cystic fibrosis transmembrane conductance regulator (CFTR) modulators correct the basic defect caused by CFTR mutations. Improvements in health outcomes have been achieved using the combination of a CFTR corrector and potentiator in people with CF (pwCF) homozygous for F508del. The addit...

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Publicado no:Lancet
Main Authors: Heijerman, Harry G M, McKone, Edward F, Downey, Damian G, Van Braeckel, Eva, Rowe, Steven M, Tullis, Elizabeth, Mall, Marcus A, Welter, John J, Ramsey, Bonnie W, McKee, Charlotte M, Marigowda, Gautham, Moskowitz, Samuel M, Waltz, David, Sosnay, Patrick R, Simard, Christopher, Ahluwalia, Neil, Xuan, Fengjuan, Zhang, Yaohua, Taylor-Cousar, Jennifer L, McCoy, Karen S
Formato: Artigo
Idioma:Inglês
Publicado em: 2019
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7571408/
https://ncbi.nlm.nih.gov/pubmed/31679946
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/S0140-6736(19)32597-8
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