Bibliography
- Derich N. Targeting a genetic defect: cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis. Eur Respir Rev 2013;22(127):58-65
- Sheppard DN, Welsh MJ. Structure and function of the CFTR chloride channel. Physiol Rev 1999;79(1 Suppl):S23-45
- Welsh MJ, Smith AE. Molecular mechanisms of CFTR chloride channel dysfunction in cystic fibrosis. Cell 1993;73(7):1251-4
- Tsui LC. Cystic fibrosis gene genetic analysis consortium. Hum Mutat 2001;4:167-77
- Davies JC, Alton EW. Gene therapy for cystic fibrosis. Proc Am Thorac Soc 2010;7(6):408-14
- Alton EW, Baker A, Baker E, et al. The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep. Biomaterials 2013;34(38):10267-77
- Eckford PD, Li C, Ramjeesingh M, Bear CE. Cystic fibrosis transmembrane conductance regulator (CFTR) potentiator VX-770 (ivacaftor) opens the defective channel gate of mutant CFTR in a phosphorylation-dependent but ATP-independent manner. J Biol Chem 2012;287(44):36639-49
- O'Reilly R, Elphick HE. Development, clinical utility, and place of ivacaftor in the treatment of cystic fibrosis. Drug Des Devel Ther 2013;7:929-37
- Van Goor F, Straley KS, Cao D, et al. Rescue of delta 508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small molecules. Am J Physiol 2006;290:L1117-30
- Van Goor F, Hadida S, Grootenhuis PD, et al. Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809. Proc Natl Acad Sci USA 2011;108(46):18843-8
- Pedemonte N, Sonawane ND, Taddei A, et al. Phenylglycine and sulfonamide correctors of defective delta F508 and G551D cystic fibrosis transmembrane conductance regulator chloride-channel gating. Mol Pharmacol 2005;67:1797-807
- Yoo CL, Yu GJ, Yang B, et al. 4'-Methyl-4,5'-bithiazole-based correctors of defective delta F508-CFTR cellular processing. Bioorg Med Chem Lett 2008;18(8):2610-14
- Donald MB, Rodriguez KX, Shay H, et al. Click-based synthesis of triazolobithiazole delta 508-CFTR correctors for cystic fibrosis. Bioorg Med Chem 2012;20(17):5247-53
- Clancy JP, Rowe SM, Accurso FJ, et al. 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 2012;67(1):12-18
- Novartis AG. Pyridine and pyrazine derivative for the treatment of CF. WO113894; 2011
- Galapagos NV. Method for identifying compounds useful for increasing the functional activity and cell surface expression of Cf-associated mutant cystic fibrosis transmembrane conductance regulator. WO029118; 2010
- Galapagos NV, R&D Update. 2013. Available from: www.glpg.com/index.php/download_file/view/1440/1/
- Galapagos NV. AbbVie and Galapagos to co-develop cystic fibrosis therapies. 2013. Available from: http://www.glpg.com/index.php/download_file/view/1619/164/
- Sun X, Sui J, Qiu J, et al. Available from: http://www.n30pharma.com/docs/posters/2013-10-15-NACFC-Sun-poster.pdf
- Novartis AG. Pyridine amide derivatives. WO038373; 2013
- Novartis AG. Pyridine amide derivatives. WO038378; 2013
- Novartis AG. Pyridine/pyrazine amide derivatives. WO03838; 2013
- Novartis AG. Heterocyclic compounds for the treatment of cystic fibrosis. WO038386; 2013
- Novartis AG. N-substituted heterocyclyl carboxamides. WO038390; 2013
- Hunt T, Atherton-Watson HC, Axford J, et al. Discovery of a novel chemotype of potent human ENaC blockers using a bioisostere approach. Part 1: quaternary amines. Bioorg Med Chem Lett 2012;22(2):929-32
- Van Goor F, Hadida S, Grootenhuis PD, et al. Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809. Proc Natl Acad Sci USA 2011;108(46):18843-87