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2021 6
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Page 1
Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele.
Middleton PG, Mall MA, Dřevínek P, Lands LC, McKone EF, Polineni D, Ramsey BW, Taylor-Cousar JL, Tullis E, Vermeulen F, Marigowda G, McKee CM, Moskowitz SM, Nair N, Savage J, Simard C, Tian S, Waltz D, Xuan F, Rowe SM, Jain R; VX17-445-102 Study Group. Middleton PG, et al. N Engl J Med. 2019 Nov 7;381(19):1809-1819. doi: 10.1056/NEJMoa1908639. Epub 2019 Oct 31. N Engl J Med. 2019. PMID: 31697873 Free PMC article. Clinical Trial.
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.
Heijerman HGM, McKone EF, Downey DG, Van Braeckel E, Rowe SM, Tullis E, Mall MA, Welter JJ, Ramsey BW, McKee CM, Marigowda G, Moskowitz SM, Waltz D, Sosnay PR, Simard C, Ahluwalia N, Xuan F, Zhang Y, Taylor-Cousar JL, McCoy KS; VX17-445-103 Trial Group. Heijerman HGM, et al. Lancet. 2019 Nov 23;394(10212):1940-1948. doi: 10.1016/S0140-6736(19)32597-8. Epub 2019 Oct 31. Lancet. 2019. PMID: 31679946 Free PMC article. Clinical Trial.
VX-445-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles.
Keating D, Marigowda G, Burr L, Daines C, Mall MA, McKone EF, Ramsey BW, Rowe SM, Sass LA, Tullis E, McKee CM, Moskowitz SM, Robertson S, Savage J, Simard C, Van Goor F, Waltz D, Xuan F, Young T, Taylor-Cousar JL; VX16-445-001 Study Group. Keating D, et al. N Engl J Med. 2018 Oct 25;379(17):1612-1620. doi: 10.1056/NEJMoa1807120. Epub 2018 Oct 18. N Engl J Med. 2018. PMID: 30334692 Free PMC article. Clinical Trial.
Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis.
Berkers G, van Mourik P, Vonk AM, Kruisselbrink E, Dekkers JF, de Winter-de Groot KM, Arets HGM, Marck-van der Wilt REP, Dijkema JS, Vanderschuren MM, Houwen RHJ, Heijerman HGM, van de Graaf EA, Elias SG, Majoor CJ, Koppelman GH, Roukema J, Bakker M, Janssens HM, van der Meer R, Vries RGJ, Clevers HC, de Jonge HR, Beekman JM, van der Ent CK. Berkers G, et al. Among authors: van der meer r. Cell Rep. 2019 Feb 12;26(7):1701-1708.e3. doi: 10.1016/j.celrep.2019.01.068. Cell Rep. 2019. PMID: 30759382 Free article.
Lumacaftor/ivacaftor in people with cystic fibrosis with an A455E-CFTR mutation.
Berkers G, van der Meer R, Heijerman H, Beekman JM, Boj SF, Vries RGJ, van Mourik P, Doyle JR, Audhya P, Yuan ZJ, Kinnman N, van der Ent CK. Berkers G, et al. Among authors: van der meer r. J Cyst Fibros. 2020 Nov 25:S1569-1993(20)30910-3. doi: 10.1016/j.jcf.2020.11.007. Online ahead of print. J Cyst Fibros. 2020. PMID: 33249003 Free article.
CFTR Modulators: Does One Dose Fit All?
van der Meer R, Wilms EB, Heijerman HGM. van der Meer R, et al. J Pers Med. 2021 May 24;11(6):458. doi: 10.3390/jpm11060458. J Pers Med. 2021. PMID: 34073663 Free PMC article. Review.
Forskolin-induced swelling of intestinal organoids correlates with disease severity in adults with cystic fibrosis and homozygous F508del mutations.
de Winter-de Groot KM, Berkers G, Marck-van der Wilt REP, van der Meer R, Vonk A, Dekkers JF, Geerdink M, Michel S, Kruisselbrink E, Vries R, Clevers H, Vleggaar FP, Elias SG, Heijerman HGM, van der Ent CK, Beekman JM. de Winter-de Groot KM, et al. Among authors: van der meer r. J Cyst Fibros. 2020 Jul;19(4):614-619. doi: 10.1016/j.jcf.2019.10.022. Epub 2019 Nov 15. J Cyst Fibros. 2020. PMID: 31735562
Clinical effects of the three CFTR potentiator treatments curcumin, genistein and ivacaftor in patients with the CFTR-S1251N gating mutation.
Berkers G, van der Meer R, van Mourik P, Vonk AM, Kruisselbrink E, Suen SW, Heijerman HG, Majoor CJ, Koppelman GH, Roukema J, Janssens HM, de Rijke YB, Kemper EM, Beekman JM, van der Ent CK, de Jonge HR. Berkers G, et al. Among authors: van der meer r. J Cyst Fibros. 2020 Nov;19(6):955-961. doi: 10.1016/j.jcf.2020.04.014. Epub 2020 Jun 1. J Cyst Fibros. 2020. PMID: 32499204
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