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
Developmental control of CFTR: from bioinformatics to novel therapeutic approaches Source: Eur Respir J 2015; 45: 18-20 Year: 2015
ENaC inhibition in cystic fibrosis: potential role in the new era of CFTR modulator therapies Source: Eur Respir J, 56 (6) 2000946; 10.1183/13993003.00946-2020 Year: 2020
Pathophysiology of CF and CFTR directed therapies Source: ERS course 2015 Year: 2015
The cystic fibrosis airway milieu enhances rescue of F508del in a pre-clinical model Source: Eur Respir J, 52 (6) 1801133; 10.1183/13993003.01133-2018 Year: 2018
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
Ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation Source: Eur Respir Rev 2013; 22: 66-71 Year: 2013
Targeting a genetic defect: cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis Source: Eur Respir Rev 2013; 22: 58-65 Year: 2013
CFTR targeted therapies: recent advances in cystic fibrosis and possibilities in other diseases of the airways Source: Eur Respir Rev, 29 (156) 190068; 10.1183/16000617.0068-2019 Year: 2020
Allosteric inhibition of the cystic fibrosis transmembrane conductance regulator (CFTR) slows airway mucus transport in normal sheep Source: International Congress 2015 – Cystic fibrosis: physiology, biomarkers, microbiology and miscellaneous Year: 2015
Effects of the CFTR potentiator, ivacaftor, in two phase 3 trials in subjects with CF who have the G551D-CFTR mutation Source: Annual Congress 2012 - Cystic fibrosis (adults and children): new therapies and detection of early lung disease Year: 2012
How organoids helped to discover cystic fibrosis transmembrane conductance regulator (CFTR) therapeutics and predict response to treatment Source: International Congress 2016 – Closing the gap: novel translational research strategies Year: 2016
A European regulatory perspective on cystic fibrosis: current treatments, trends in drug development and translational challenges for CFTR modulators Source: Eur Respir Rev, 27 (148) 170124; 10.1183/16000617.0124-2017 Year: 2018
ADME properties of a novel PI3Kd inhibitor, DS-1515, in preclinical studies Source: International Congress 2018 – Basic pharmacology Year: 2018
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
The era of CFTR modulators: improvements made and remaining challenges Source: Breathe, 16 (2) 200016; 10.1183/20734735.0016-2020 Year: 2020
Novel therapeutics for CF Source: International Congress 2019 – Future of cystic fibrosis care: insights from the Lancet Commission Year: 2019
CFTR dysfunction and its relationship to phenotype and development of new treatments restoring CFTR function Source: Annual Congress 2013 –PG20 Cystic fibrosis: an update of the basic defects and clinical problems in CF children and adults Year: 2013