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Effectiveness of ivacaftor in cystic fibrosis Patients with non-G551D gating mutations

BACKGROUND: The cystic fibrosis transmembrane conductance regulator (CFTR) potentiator ivacaftor is approved for patients with CF with gating and residual function CFTR mutations. We report the results of an observational study investigating its effects in CF patients with non-G551D gating mutations...

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Detalhes bibliográficos
Publicado no:J Cyst Fibros
Main Authors: Guimbellot, Jennifer, Solomon, George M., Baines, Arthur, Heltshe, Sonya L., VanDalfsen, Jill, Joseloff, Elizabeth, Sagel, Scott D., Rowe, Steven M.
Formato: Artigo
Idioma:Inglês
Publicado em: 2018
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6196121/
https://ncbi.nlm.nih.gov/pubmed/29685811
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.jcf.2018.04.004
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