CFTR modulators for cystic fibrosis

Journal of the American Academy of Physician Assistants

Volumn 26, Issue 2, 2013, Pages 59-60

  Bertoncini, Erica ^a   Colomb Lippa, Dawn ^a  

^a NONE

Author keywords

[No Author keywords available]

Indexed keywords

1,3 BENZODIOXOLE DERIVATIVE; AMINOPHENOL DERIVATIVE; AMINOPYRIDINE DERIVATIVE; ATALUREN; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; IVACAFTOR; LUMACAFTOR; N-(2,4-DI-TERT-BUTYL-5-HYDROXYPHENYL)-4-OXO-1,4-DIHYDROQUINOLINE-3-CARBOXAMIDE; OXADIAZOLE DERIVATIVE; QUINOLONE DERIVATIVE;

ARTICLE; CYSTIC FIBROSIS; DRUG EFFECT; GENETICS; HUMAN; LUNG; MUTATION; PATHOPHYSIOLOGY;

AMINOPHENOLS; AMINOPYRIDINES; BENZODIOXOLES; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; HUMANS; LUNG; MUTATION; OXADIAZOLES; QUINOLONES;

MLCS; MLOWN;

EID: 84875760563     PISSN: 15471896     EISSN: 08937400     Source Type: Journal    
DOI: 10.1097/01720610-201302000-00013     Document Type: Note

References (14)

1. Cystic Fibrosis Foundation. About 65 roses. http://www.cff.org/aboutCFFoundation/About65Roses/. Updated September 19, 2006. Accessed January 10, 2013.
2. Cystic Fibrosis Foundation. Cystic Fibrosis Foundation Patient Registry Annual Data Report 2010. http://www.cff.org/UploadedFiles/LivingWithCF/CareCenterNetwork/PatientRegistry/2010-Patient-Registry-Report.pdf. Published 2011. Accessed January 10, 2013.
3. Cystic Fibrosis Foundation. Drug development pipeline. http://www.cff.org/research/DrugDevelopmentPipeline/. Accessed January 10, 2013.
4. Riordan JR, Rommens JM, Kerem B, et al. Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA. Science. 1989;245(4922):1066-1073.
5. Knowles MR, Stutts MJ, Spock A, et al. Abnormal ion permeation through cystic fibrosis respiratory epithelium. Science. 1983;221(4615):1067-1070.
6. Ramsey BW, Davies J, McElvaney NG, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365(18):1663-1672.
7. Van Goor F, Hadida S, Grootenhuis PD, et al. Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770. Proc Natl Acad Sci USA. 2009;106(44):18825-18830.
8. Sermet-Gaudelus I, Boeck KD, Casimir GJ, et al. Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis. Am J Respir Crit Care Med. 2010;182(10):1262-1272.
9. Van Goor F, Hadida S, Grootenhuis P, 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):18843-18848.
10. Wilschanski M, Miller LL, Shoseyov D, et al. Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis. Eur Respir J. 2011;38(1):59-69.
11. 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.
12. Flume PA, O'Sullivan BP, Robinson KA, et al. Cystic fibrosis pulmonary guidelines: chronic medications for maintenance of lung health. Am J Respir Crit Care Med. 2007;176(10):957-969.
13. Flume PA, Mogayzel PJ, Robinson KA, et al. Cystic fibrosis pulmonary guidelines: Treatment of pulmonary exacerbations. Am J Respir Crit Care Med. 2009;180(9):802-808.
14. Yankaskas JR, Mallory GB Jr. Lung transplantation in cystic fibrosis. Chest. 1998;113(1):217-226.