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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Gepubliceerd in:J Cyst Fibros
Hoofdauteurs: Guimbellot, Jennifer, Solomon, George M., Baines, Arthur, Heltshe, Sonya L., VanDalfsen, Jill, Joseloff, Elizabeth, Sagel, Scott D., Rowe, Steven M.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2018
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Online toegang: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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