Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR

Rationale: Tezacaftor (formerly VX-661) is an investigational small molecule that improves processing and trafficking of the cystic fibrosis transmembrane conductance regulator (CFTR) in vitro, and improves CFTR function alone and in combination with ivacaftor. Objectives: To evaluate the safety and...

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Publicado en:Am J Respir Crit Care Med
Autores principales: Donaldson, Scott H., Pilewski, Joseph M., Griese, Matthias, Cooke, Jon, Viswanathan, Lakshmi, Tullis, Elizabeth, Davies, Jane C., Lekstrom-Himes, Julie A., Wang, Linda T.
Formato: Artigo
Lenguaje:Inglês
Publicado: American Thoracic Society 2018
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Original Articles
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC5768901/
https://ncbi.nlm.nih.gov/pubmed/28930490
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1164/rccm.201704-0717OC
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