Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation - Clancy, J P; Rowe, Steven M; Accurso, Frank J; Aitken, Moira L; Amin, Raouf S; Ashlock, Melissa A; Ballmann, Manfred; Boyle, Michael P; Bronsveld, Inez; Campbell, Preston W; De Boeck, Kris; Donaldson, Scott H; et al. (Thorax, 2012)
Keywords: Adolescent; Adult; Aminopyridines - administration & dosage; Aminopyridines - pharmacokinetics; Benzodioxoles - administration & dosage; Benzodioxoles - pharmacokinetics; Chloride; Cystic fibrosis; Cystic Fibrosis - drug therapy; Cystic Fibrosis - genetics; Cystic Fibrosis - metabolism; Cystic Fibrosis Transmembrane Conductance Regulator - drug effects; Cystic Fibrosis Transmembrane Conductance Regulator - genetics; Cystic Fibrosis Transmembrane Conductance Regulator - metabolism; DNA - genetics; DNA Mutational Analysis; Dose-Response Relationship, Drug; Double-Blind Method; Drug dosages; Female; Follow-Up Studies; Homozygote; Humans; Lung diseases; Male; Middle Aged; Mutation; Prospective Studies; Proteins; Sweat Glands - metabolism; Treatment Outcome; White people; Young Adult