Ivacaftor in a G551D homozygote with cystic fibrosis

New England Journal of Medicine

Volumn 369, Issue 13, 2013, Pages 1280-1282

  Harrison, Michael J ^a   Murphy, Desmond M ^a   Plant, Barry J ^a  

^a UNIVERSITY COLLEGE CORK   (Ireland)

Author keywords

[No Author keywords available]

Indexed keywords

ALBUMIN; ANTIBIOTIC AGENT; CHLORIDE; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; IVACAFTOR;

ADULT; ALBUMIN BLOOD LEVEL; CASE REPORT; CYSTIC FIBROSIS; DISEASE EXACERBATION; DRUG EFFICACY; FEMALE; FORCED EXPIRATORY VOLUME; GENE DELETION; GENE MUTATION; HETEROZYGOSITY; HOMOZYGOTE; HUMAN; LETTER; LUNG FUNCTION; LUNG TRANSPLANTATION; MULTIPLE CYCLE TREATMENT; OXYGEN SATURATION; PREDICTIVE VALUE; PREVALENCE; PRIORITY JOURNAL; WALKING SPEED;

EID: 84884517511     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMc1213681     Document Type: Letter

References (5)

1. 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;365:1663-72.
2. Rowe SM, Miller S, Sorscher EJ. Cystic fibrosis. N Engl J Med 2005;352:1992-2001. (Pubitemid 40638370)
3. Polenakovik HM, Sanville B. The use of ivacaftor in an adult with severe lung disease due to cystic fibrosis (ΔF508/G551D). J Cyst Fibros (in press).
4. Fletcher G. Cystic Fibrosis Registry of Ireland, Annual Report 2010 (http://www.cfri.ie/docs/annual-reports/CFRI2010.pdf).
5. Marshall BC. Cystic Fibrosis Foundation patient registry: annual data report 2010 (http://www.cff.org/UploadedFiles/LivingWithCF/CareCenterNetwork/ PatientRegistry/2010-Patient -Registry-Report.pdf).