Clinical Mechanism of the Cystic Fibrosis Transmembrane Conductance Regulator Potentiator Ivacaftor...
Clinical Mechanism of the Cystic Fibrosis Transmembrane Conductance Regulator Potentiator Ivacaftor in G551D-mediated Cystic Fibrosis
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Author / Creator
GOAL Investigators of the Cystic Fibrosis Foundation Therapeutics Development Network , 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 and Ramsey, Bonnie W.
Publisher
United States: American Thoracic Society
Journal title
Language
English
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Publication information
Publisher
United States: American Thoracic Society
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Scope and Contents
Contents
Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator recently approved for patients with CF age 6 and older with the G551D mutation.
To evaluate ivacaftor in a postapproval setting and determine mechanism of action and response of clinically relevant markers.
We conducted a longitudinal cohort study in 2012-20...
Alternative Titles
Full title
Clinical Mechanism of the Cystic Fibrosis Transmembrane Conductance Regulator Potentiator Ivacaftor in G551D-mediated Cystic Fibrosis
Authors, Artists and Contributors
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Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4226057
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4226057
Other Identifiers
ISSN
1073-449X
E-ISSN
1535-4970
DOI
10.1164/rccm.201404-0703OC
