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VX-659-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles.
Davies JC, Moskowitz SM, Brown C, Horsley A, Mall MA, McKone EF, Plant BJ, Prais D, Ramsey BW, Taylor-Cousar JL, Tullis E, Uluer A, McKee CM, Robertson S, Shilling RA, Simard C, Van Goor F, Waltz D, Xuan F, Young T, Rowe SM; VX16-659-101 Study Group. Davies JC, et al. N Engl J Med. 2018 Oct 25;379(17):1599-1611. doi: 10.1056/NEJMoa1807119. Epub 2018 Oct 18. N Engl J Med. 2018. PMID: 30334693 Free PMC article. Clinical Trial.
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).
Southern KW, Murphy J, Sinha IP, Nevitt SJ. Southern KW, et al. Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3. Cochrane Database Syst Rev. 2020. Update in: Cochrane Database Syst Rev. 2023 Nov 20;11:CD010966. doi: 10.1002/14651858.CD010966.pub4. PMID: 33331662 Free PMC article. Updated.