VX-659-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles

BACKGROUND: The next-generation cystic fibrosis transmembrane conductance regulator (CFTR) corrector VX-659, in triple combination with tezacaftor and ivacaftor (VX-659-tezacaftor-ivacaftor), was developed to restore the function of Phe508del CFTR protein in patients with cystic fibrosis. METHODS: W...

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Detalhes bibliográficos
Publicado no:N Engl J Med
Main Authors: Davies, Jane C., Moskowitz, Samuel M., Brown, Cynthia, Horsley, Alexander, Mall, Marcus A., McKone, Edward F., Plant, Barry J., Prais, Dario, Ramsey, Bonnie W., Taylor-Cousar, Jennifer L., Tullis, Elizabeth, Uluer, Ahmet, McKee, Charlotte M., Robertson, Sarah, Shilling, Rebecca A., Simard, Christopher, Van Goor, Fredrick, Waltz, David, Xuan, Fengjuan, Young, Tim, Rowe, Steven M.
Formato: Artigo
Idioma:Inglês
Publicado em: 2018
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6277022/
https://ncbi.nlm.nih.gov/pubmed/30334693
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa1807119
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