Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del / F508del-CFTR or F508del / G551D-CFTR - Donaldson, Scott H.; Pilewski, Joseph M.; Griese, Matthias; Cooke, Jon; Viswanathan, Lakshmi; Tullis, Elizabeth; Davies, Jane C.; Lekstrom-Himes, Julie A.; Wang, Linda T. (American journal of respiratory and critical care medicine, 2018)
Keywords: Administration, Oral; Adolescent; Adult; Aminophenols - administration & dosage; Aminophenols - adverse effects; Benzodioxoles - administration & dosage; Benzodioxoles - adverse effects; Chloride; Cystic fibrosis; Cystic Fibrosis - diagnosis; Cystic Fibrosis - drug therapy; Cystic Fibrosis - genetics; Cystic Fibrosis Transmembrane Conductance Regulator - genetics; Cysts; Dose-Response Relationship, Drug; Double-Blind Method; Drug Administration Schedule; Drug dosages; Drug Therapy, Combination; Female; Humans; Indoles - administration & dosage; Indoles - adverse effects; Kinases; Male; Maximum Tolerated Dose; Molecular Targeted Therapy - methods; Mutation; Original; Patients; Pediatrics; Prognosis; Proteins; Quinolones - administration & dosage; Quinolones - adverse effects; Respiratory Function Tests; Risk Assessment; Severity of Illness Index; Treatment Outcome; Young Adult; United States--US; forced expiratory volume; sweat chloride; CFTR modulator; cystic fibrosis transmembrane conductance regulator corrector