CFTR modulator therapy for cystic fibrosis

New England Journal of Medicine

Volumn 377, Issue 21, 2017, Pages 2085-2088

  Grasemann, Hartmut ^a  

^a HOSPITAL FOR SICK CHILDREN   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

ANTIBIOTIC AGENT; ASPARTIC ACID; GLYCINE; IVACAFTOR; IVACAFTOR PLUS LUMACAFTOR; IVACAFTOR PLUS TEZACAFTOR; LUMACAFTOR; MUCOLYTIC AGENT; PLACEBO; RECOMBINANT ENZYME; RECOMBINANT HUMAN DEOXYRIBONUCLEASE; TEZACAFTOR; UNCLASSIFIED DRUG; AMINOPHENOL DERIVATIVE; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR;

ALLELE; CELL SURFACE; CELL TRANSPORT; CFTR DEFICIENCY; CFTR GENE; COMBINATION CHEMOTHERAPY; CYSTIC FIBROSIS; DRUG EFFICACY; DRUG POTENTIATION; DYSPNEA; EDITORIAL; FORCED EXPIRATORY VOLUME; GENE DELETION; GENE DELIVERY SYSTEM; GENE FUNCTION; GENETIC ASSOCIATION; GENOTYPE; HEALTH STATUS; HETEROZYGOSITY; HOMOZYGOSITY; HUMAN; LIVER INJURY; LUNG DISEASE; LUNG FUNCTION; MONOTHERAPY; PATHOGENESIS; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; PROTEIN DEFICIENCY; PROTEIN FUNCTION; QUALITY OF LIFE; RESIDUAL VOLUME; STEM CELL TRANSPLANTATION; TREATMENT DURATION; GENETICS; MUTATION;

AMINOPHENOLS; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; HUMANS; MUTATION;

EID: 85034779796     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMe1712335     Document Type: Editorial

References (9)

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. 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.
3. Boyle MP, Bell SC, Konstan MW, et al. A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial. Lancet Respir Med 2014;2:527-38.
4. Wainwright CE, Elborn JS, Ramsey BW, et al. Lumacaftor–ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR. N Engl J Med 2015;373:220-31.
5. Veit G, Avramescu RG, Perdomo D, et al. Some gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression. Sci Transl Med 2014;6:246ra97.
6. Taylor-Cousar JL, Munck A, McKone EF, et al. Tezacaftor–ivacaftor in patients with cystic fibrosis homozygous for Phe508del. N Engl J Med 2017;377:2013-23.
7. Rowe SM, Daines C, Ringshausen FC, et al. Tezacaftor–ivacaftor in residual-function heterozygotes with cystic fibrosis. N Engl J Med 2017;377:2024-35.
8. Drug development pipeline: we’re attacking CF from every angle. Bethesda, MD: Cystic Fibrosis Foundation (https://www.cff.org/Trials/Pipeline).
9. Vertex announces positive Phase 1 & Phase 2 data from three different triple combination regimens in people with cystic fibrosis who have one F508del mutation and one minimal function mutation (F508del/Min). Press release of Vertex, July 18, 2017 (http://investors.vrtx.com/releasedetail.cfm?ReleaseID=1033559).