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Long term clinical effectiveness of ivacaftor in people with the G551D CFTR mutation

BACKGROUND: The cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, ivacaftor, was first approved for people with CF and the G551D CFTR mutation. This study describes the long-term clinical effectiveness of ivacaftor in this population. METHODS: We conducted a multicenter, prospe...

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Detalhes bibliográficos
Publicado no:J Cyst Fibros
Main Authors: Guimbellot, J.S., Baines, A., Paynter, A., Heltshe, S.L., VanDalfsen, J., Jain, M., Rowe, S.M., Sagel, S.D.
Formato: Artigo
Idioma:Inglês
Publicado em: 2020
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC8183611/
https://ncbi.nlm.nih.gov/pubmed/33249004
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.jcf.2020.11.008
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