Clinical Mechanism of the Cystic Fibrosis Transmembrane Conductance Regulator Potentiator Ivacaftor in G551D-mediated Cystic Fibrosis

Rationale: Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator recently approved for patients with CF age 6 and older with the G551D mutation. Objectives: To evaluate ivacaftor in a postapproval setting and determine mechanism of action and response of clinically re...

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Veröffentlicht in:Am J Respir Crit Care Med
Hauptverfasser: Rowe, Steven M., Heltshe, Sonya L., Gonska, Tanja, Donaldson, Scott H., Borowitz, Drucy, Gelfond, Daniel, Sagel, Scott D., Khan, Umer, Mayer-Hamblett, Nicole, Van Dalfsen, Jill M., Joseloff, Elizabeth, Ramsey, Bonnie W.
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Thoracic Society 2014
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Original Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4226057/
https://ncbi.nlm.nih.gov/pubmed/24927234
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1164/rccm.201404-0703OC
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