Change in sweat chloride as a clinical end point in cystic fibrosis clinical trials: the ivacaftor experience
AG Durmowicz, KA Witzmann, CJ Rosebraugh… - Chest, 2013 - Elsevier
Cystic fibrosis (CF) is a life-shortening inherited disease caused by mutations in the CF
transmembrane conductance regulator gene (CFTR), which encodes for the CF
transmembrane conductance regulator (CFTR) ion channel that regulates chloride and
water transport across the surface of epithelial cells. Ivacaftor, a drug recently approved by
the US Food and Drug Administration, represents the first mutation-specific therapy for CF. It
is a CFTR channel modulator and improves CFTR function in patients with CF who have a …
transmembrane conductance regulator gene (CFTR), which encodes for the CF
transmembrane conductance regulator (CFTR) ion channel that regulates chloride and
water transport across the surface of epithelial cells. Ivacaftor, a drug recently approved by
the US Food and Drug Administration, represents the first mutation-specific therapy for CF. It
is a CFTR channel modulator and improves CFTR function in patients with CF who have a …