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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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Publicat a:J Cyst Fibros
Autors principals: Guimbellot, Jennifer, Solomon, George M., Baines, Arthur, Heltshe, Sonya L., VanDalfsen, Jill, Joseloff, Elizabeth, Sagel, Scott D., Rowe, Steven M.
Format: Artigo
Idioma:Inglês
Publicat: 2018
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Accés en línia: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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