elexacaftor
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Indications
- cystic fibrosis with Phe508del allele
More general terms
References
- ↑ Middleton PG, Mall MA, Drevinek P et al. Elexacaftor-tezacaftor-ivacaftor for cystic fibrosis with a single Phe508del allele. N Engl J Med 2019 Nov 7; PMID: https://www.ncbi.nlm.nih.gov/pubmed/31697873 https://www.nejm.org/doi/10.1056/NEJMoa1908639
Heijerman HGM, McKone EF, Downey EG et al. Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: A double-blind, randomised, phase 3 trial. Lancet 2019 Oct 30 PMID: https://www.ncbi.nlm.nih.gov/pubmed/31679946 https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(19)32597-8/fulltext