Lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR

New England Journal of Medicine

Volumn 373, Issue 18, 2015, Pages 1783-

  Rehman, Abdul ^a   Ul Ain Baloch, Noor ^b   Janahi, Ibrahim A ^a  

^a HAMAD MEDICAL CORPORATION   (Qatar)

^b AGA KHAN UNIVERSITY   (Pakistan)

Author keywords

[No Author keywords available]

Indexed keywords

AZITHROMYCIN; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; IBUPROFEN; IVACAFTOR PLUS LUMACAFTOR; PHENYLALANINE; SODIUM CHLORIDE; 1,3 BENZODIOXOLE DERIVATIVE; AMINOPHENOL DERIVATIVE; AMINOPYRIDINE DERIVATIVE; QUINOLONE DERIVATIVE;

CFTR GENE; COST EFFECTIVENESS ANALYSIS; CYSTIC FIBROSIS; DRUG COST; DRUG EFFICACY; FORCED EXPIRATORY VOLUME; GENE DELETION; HOMOZYGOSITY; HUMAN; LETTER; PRIORITY JOURNAL; FEMALE; GENETICS; MALE;

AMINOPHENOLS; AMINOPYRIDINES; BENZODIOXOLES; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; FEMALE; HUMANS; MALE; QUINOLONES;

EID: 84946048953     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMc1510466     Document Type: Letter

References (5)

1. Wainwright CE, Elborn JS, Ramsey BW, et al. Lumacaftor ivacaftor in patients with cystic fibrosis homozygous for Phe 508del CFTR. N Engl J Med 2015;373:220-31.
2. Davies JC, Wainwright CE, Canny GJ, et al. Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with a G551D mutation. Am J Respir Crit Care Med 2013; 187:1219-25.
3. Flume PA, Liou TG, Borowitz DS, et al. Ivacaftor in subjects with cystic fibrosis who are homozygous for the F508del-CFTR mutation. Chest 2012;142:718-24.
4. Whiting P, Al M, Burgers L, et al. Ivacaftor for the treatment of patients with cystic fibrosis and the G551D mutation: a systematic review and cost-effectiveness analysis. Southampton, United Kingdom: NIHR Journals Library, March 2014 (Health Technology Assessment, no. 18.18) (http://www.ncbi.nlm.nih.gov/books/NBK261768).
5. Bobadilla JL, Macek M Jr, Fine JP, Farrell PM. Cystic fibrosis: a worldwide analysis of CFTR mutations - correlation with incidence data and application to screening. Hum Mutat 2002; 19:575-606.