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Clinical mechanism of the cystic fibrosis transmembrane conductance regulator potentiator ivacaftor in G551D-mediated cystic fibrosis.
Steven M Rowe
1 ,
Sonya L Heltshe ,
Tanja Gonska ,
Scott H Donaldson ,
Drucy Borowitz ,
Daniel Gelfond ,
Scott D Sagel ,
Umer Khan ,
Nicole Mayer-Hamblett ,
Jill M Van Dalfsen ,
Elizabeth Joseloff ,
Bonnie W Ramsey
Jul 15 2014
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Abstract
Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator recently approved for patients with CF age 6 and older with the G551D mutation.