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Safety and efficacy of vanzacaftor-tezacaftor-deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials.
Uluer AZ, MacGregor G, Azevedo P, Indihar V, Keating C, Mall MA, McKone EF, Ramsey BW, Rowe SM, Rubenstein RC, Taylor-Cousar JL, Tullis E, Yonker LM, Chu C, Lam AP, Nair N, Sosnay PR, Tian S, Van Goor F, Viswanathan L, Waltz D, Wang LT, Xi Y, Billings J, Horsley A; VX18-121-101; VX18-561-101 Study Groups. Uluer AZ, et al. Among authors: van goor f. Lancet Respir Med. 2023 Jun;11(6):550-562. doi: 10.1016/S2213-2600(22)00504-5. Epub 2023 Feb 23. Lancet Respir Med. 2023. PMID: 36842446 Free article. Clinical Trial.
G970R-CFTR Mutation (c.2908G>C) Results Predominantly in a Splicing Defect.
Fidler MC, Buckley A, Sullivan JC, Statia M, Boj SF, Vries RGJ, Munck A, Higgins M, Moretto Zita M, Negulescu P, van Goor F, De Boeck K. Fidler MC, et al. Among authors: van goor f. Clin Transl Sci. 2021 Mar;14(2):656-663. doi: 10.1111/cts.12927. Epub 2020 Dec 6. Clin Transl Sci. 2021. PMID: 33278322 Free PMC article. Clinical Trial.
In utero and postnatal VX-770 administration rescues multiorgan disease in a ferret model of cystic fibrosis.
Sun X, Yi Y, Yan Z, Rosen BH, Liang B, Winter MC, Evans TIA, Rotti PG, Yang Y, Gray JS, Park SY, Zhou W, Zhang Y, Moll SR, Woody L, Tran DM, Jiang L, Vonk AM, Beekman JM, Negulescu P, Van Goor F, Fiorino DF, Gibson-Corley KN, Engelhardt JF. Sun X, et al. Among authors: van goor f. Sci Transl Med. 2019 Mar 27;11(485):eaau7531. doi: 10.1126/scitranslmed.aau7531. Sci Transl Med. 2019. PMID: 30918114 Free PMC article.
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. Among authors: van goor f. 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.
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. Among authors: van goor f. 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.
Defining the disease liability of variants in the cystic fibrosis transmembrane conductance regulator gene.
Sosnay PR, Siklosi KR, Van Goor F, Kaniecki K, Yu H, Sharma N, Ramalho AS, Amaral MD, Dorfman R, Zielenski J, Masica DL, Karchin R, Millen L, Thomas PJ, Patrinos GP, Corey M, Lewis MH, Rommens JM, Castellani C, Penland CM, Cutting GR. Sosnay PR, et al. Among authors: van goor f. Nat Genet. 2013 Oct;45(10):1160-7. doi: 10.1038/ng.2745. Epub 2013 Aug 25. Nat Genet. 2013. PMID: 23974870 Free PMC article.
59 results