Targeting a genetic defect: cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis Source: Eur Respir Rev 2013; 22: 58-65 Year: 2013
Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis Source: Eur Respir J 2011; 37: 59-69 Year: 2011
Forecasting US ivacaftor outcomes and cost in cystic fibrosis patients with the G551D mutation Source: Eur Respir J 2016; 47:1697-1705 Year: 2016
Clinical development of triple-combination CFTR modulators for cystic fibrosis patients with one or two F508del alleles Source: ERJ Open Res, 5 (2) 00082-2019; 10.1183/23120541.00082-2019 Year: 2019
A novel CFTR mutation found in a Chinese cystic fibrosis patient Source: Eur Respir J 2005; 26: Suppl. 49, 402s Year: 2005
Genotype/phenotype correlation of the G85E mutation in a large cohort of cystic fibrosis patients Source: Eur Respir J 2004; 23: 679-684 Year: 2004
Improved CFTR and lung function with VX-770, a novel investigational potentiator of CFTR, in subjects with the G551D-CFTR mutation Source: Annual Congress 2010 - Cystic fibrosis: new targets for therapy and impact of viral infections Year: 2010
Potentiating and correcting mutant CFTR in patients with cystic fibrosis Source: Eur Respir Monogr 2014; 64: 129-149 Year: 2014
Late-breaking abstract: VX-770, an investigational CFTR potentiator, in subjects with CF and the G551D mutation Source: Annual Congress 2011 - Cystic fibrosis: detection and monitoring of early lung disease Year: 2011
Targeting CFTR as a therapy for cystic fibrosis Source: Lung Science Conference 2010 - Biology of gene-environment interactions in the lung Year: 2010
CFTR: cystic fibrosis and beyond Source: Eur Respir J 2014; 44: 1042-1054 Year: 2014
Lung function, weight, and sweat chloride responses in patients with cystic fibrosis and the G551D-CFTR mutation treated with ivacaftor: A secondary analysis Source: Annual Congress 2013 –Cystic fibrosis: lung function and change of lung function in infants and children before and after treatment Year: 2013
Atypical cystic fibrosis Source: ISSN=ISSN 1025-448x, ISBN=ISBN 1-904097-46-6, page=38 Year: 2006
CFTR gene mutations detected in Japanese individuals with cystic fibrosis Source: Eur Respir J 2006; 28: Suppl. 50, 716s Year: 2006
High morbidity and mortality in cystic fibrosis patients compound heterozygous for 3905insT and ΔF508 Source: Eur Respir J 2001; 17: 1181-1186 Year: 2001
Combination of ENaC and CFTR mutations may predispose to cystic fibrosis-like disease Source: Eur Respir J 2009; 34: 772 Year: 2009
Identification of a new cystic fibrosis transmembrane regulator mutation in a severely affected patient Source: Eur Respir J 2002; 19: 374-376 Year: 2002
Pharmacological treatment of the biochemical defect in cystic fibrosis airways Source: Eur Respir J 2001; 17: 1314-1321 Year: 2001
Cystic fibrosis transmembrane conductance regulator pharmacotherapy Source: ISSN=ISSN 1025-448x, ISBN=ISBN 1-904097-46-6, page=88 Year: 2006