[2015-July-02] FDA approves new treatment for cystic fibrosis

[2015-July-02] FDA approves new treatment for cystic fibrosis

http://www.fda.gov/newsevents/newsroom/pressannouncements/ucm453565.htm

Orkambi.cdx

Fredrick Van Goor, Sabine Hadida, Peter D. J. Grootenhuis, Bill Burton, Dong Cao, Tim Neuberger, Amanda Turnbull, Ashvani Singh, John Joubran, Anna Hazlewood, Jinglan Zhou, Jason McCartney, Vijayalaksmi Arumugam, Caroline Decker, Jennifer Yang, Chris Young, Eric R. Olson, Jeffery J. Wine, Raymond A. Frizzell, Melissa Ashlock and Paul Negulescu, Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770, Proc. Natl. Acad. Sci. U. S. A., 2009, 106 (44), pp 18825-18830

http://dx.doi.org/10.1073/pnas.0904709106

Fredrick Van Goor, Sabine Hadida, Peter D. J. Grootenhuis, Bill Burton, Jeffrey H. Stack, Kimberly S. Straley, Caroline J. Decker, Mark Miller, Jason McCartney, Eric R. Olson, Jeffrey J. Wine, Ray A. Frizzell, Melissa Ashlock, and Paul A. Negulescu, Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809, Proc. Natl. Acad. Sci. U. S. A., 2011, 108 (46), pp 18843-18848

http://dx.doi.org/10.1073/pnas.1105787108

Steven M. Rowe, Stacey Miller, and Eric J. Sorscher, Cystic Fibrosis, N. Engl. J. Med., 2005, 352 (19), pp 1992-2001

http://dx.doi.org/10.1056/NEJMra043184

Pamela B. Davis, Cystic Fibrosis Since 1938, Am. J. Respir. Crit. Care Med., 2006, 173 (5), pp 475-482

http://dx.doi.org/10.1164/rccm.200505-840OE

William B. Guggino and Bruce A. Stanton, Mechanisms of disease: New insights into cystic fibrosis: molecular switches that regulate CFTR, Nat. Rev. Mol. Cell Biol., 2006, 7 (6), pp 426-436

http://dx.doi.org/10.1038/nrm1949

Zhi-wei Cai, Jia Liu, Hong-yu Li and David N. Sheppard, Targeting F508del-CFTR to develop rational new therapies for cystic fibrosis, Acta Pharmacol. Sin., 2011, 32 (6), pp 693–701

http://dx.doi.org/10.1038/aps.2011.71

Taylor Sitarik Cohen and Alice Prince, Cystic fibrosis: a mucosal immunodeficiency syndrome, Nat. Med., 2012, 18 (4), pp 509–519

http://dx.doi.org/10.1038/nm.2715

Ruth O'Reilly and Heather E. Elphick, Development, clinical utility, and place of ivacaftor in the treatment of cystic fibrosis, Drug Des. Dev. Ther., 2013, (7), pp 929-937

http://dx.doi.org/10.2147/DDDT.S30345