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
About this item
Full title
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
Formats
Publication information
Publisher
United States: American Thoracic Society
Subjects
More information
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
Author / Creator
Identifiers
Primary Identifiers
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
