Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation

Boucher, 2007, Airway surface dehydration in cystic fibrosis: pathogenesis and therapy, Annu Rev Med, 58, 157, 10.1146/annurev.med.58.071905.105316 Boyle, 2013, A new era in the treatment of cystic fibrosis: correction of the underlying CFTR defect, Lancet Respir Med, 1, 158, 10.1016/S2213-2600(12)70057-7 Clancy, 2012, 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, Thorax, 67, 12, 10.1136/thoraxjnl-2011-200393 The Clinical Functional TRanslation of CFTR (CFTR2) Davies, 2013, Efficacy and safety of ivacaftor in patients aged 6 to 11years with cystic fibrosis with a G551D mutation, Am J Respir Crit Care Med, 187, 1219, 10.1164/rccm.201301-0153OC De Boeck, 2014, The relative frequency of CFTR mutation classes in European patients with cystic fibrosis, J Cyst Fibros, 13, 403, 10.1016/j.jcf.2013.12.003 Fuchs, 1994, Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. The Pulmozyme Study Group, N Engl J Med, 331, 637, 10.1056/NEJM199409083311003 Hankinson, 1999, Spirometric reference values from a sample of the general U.S. population, Am J Respir Crit Care Med, 159, 179, 10.1164/ajrccm.159.1.9712108 Hwang, 2009, Gating of the CFTR Cl- channel by ATP-driven nucleotide-binding domain dimerisation, J Physiol, 587, 2151, 10.1113/jphysiol.2009.171595 Konstan, 2010, Trends in the use of routine therapies in cystic fibrosis: 1995–2005, Pediatr Pulmonol, 45, 1167, 10.1002/ppul.21315 Quittner, 2005, Development and validation of the Cystic Fibrosis Questionnaire in the United States: a health-related quality-of-life measure for cystic fibrosis, Chest, 128, 2347, 10.1378/chest.128.4.2347 Ramsey, 2011, A CFTR potentiator in patients with cystic fibrosis and the G551D mutation, N Engl J Med, 365, 1663, 10.1056/NEJMoa1105185 Riordan, 1989, Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA, Science, 245, 1066, 10.1126/science.2475911 Rosenstein, 1998, The diagnosis of cystic fibrosis: a consensus statement. Cystic Fibrosis Foundation Consensus Panel, J Pediatr, 132, 589, 10.1016/S0022-3476(98)70344-0 Rowe, 2005, Cystic fibrosis, N Engl J Med, 352, 1992, 10.1056/NEJMra043184 Rubenstein, 2006, Targeted therapy for cystic fibrosis: cystic fibrosis transmembrane conductance regulator mutation-specific pharmacologic strategies, Mol Diagn Ther, 10, 293, 10.1007/BF03256204 Van Goor, 2009, Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770, Proc Natl Acad Sci USA, 106, 18825, 10.1073/pnas.0904709106 Viviani, 2012 Wang, 1993, Pulmonary function between 6 and 18years of age, Pediatr Pulmonol, 15, 75, 10.1002/ppul.1950150204 Yu, 2012, Ivacaftor potentiation of multiple CFTR channels with gating mutations, J Cyst Fibros, 11, 237, 10.1016/j.jcf.2011.12.005