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Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation.
Clancy JP, Rowe SM, Accurso FJ, Aitken ML, Amin RS, Ashlock MA, Ballmann M, Boyle MP, Bronsveld I, Campbell PW, De Boeck K, Donaldson SH, Dorkin HL, Dunitz JM, Durie PR, Jain M, Leonard A, McCoy KS, Moss RB, Pilewski JM, Rosenbluth DB, Rubenstein RC, Schechter MS, Botfield M, Ordoñez CL, Spencer-Green GT, Vernillet L, Wisseh S, Yen K, Konstan MW. Clancy JP, et al. Among authors: de boeck k. Thorax. 2012 Jan;67(1):12-8. doi: 10.1136/thoraxjnl-2011-200393. Epub 2011 Aug 8. Thorax. 2012. PMID: 21825083 Free PMC article. Clinical Trial.
Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.
Kerem E, Konstan MW, De Boeck K, Accurso FJ, Sermet-Gaudelus I, Wilschanski M, Elborn JS, Melotti P, Bronsveld I, Fajac I, Malfroot A, Rosenbluth DB, Walker PA, McColley SA, Knoop C, Quattrucci S, Rietschel E, Zeitlin PL, Barth J, Elfring GL, Welch EM, Branstrom A, Spiegel RJ, Peltz SW, Ajayi T, Rowe SM; Cystic Fibrosis Ataluren Study Group. Kerem E, et al. Among authors: de boeck k. Lancet Respir Med. 2014 Jul;2(7):539-47. doi: 10.1016/S2213-2600(14)70100-6. Epub 2014 May 15. Lancet Respir Med. 2014. PMID: 24836205 Free PMC article. Clinical Trial.
Diagnosis of Cystic Fibrosis in Nonscreened Populations.
Sosnay PR, White TB, Farrell PM, Ren CL, Derichs N, Howenstine MS, Nick JA, De Boeck K. Sosnay PR, et al. Among authors: de boeck k. J Pediatr. 2017 Feb;181S:S52-S57.e2. doi: 10.1016/j.jpeds.2016.09.068. J Pediatr. 2017. PMID: 28129813
Antisense oligonucleotide eluforsen improves CFTR function in F508del cystic fibrosis.
Sermet-Gaudelus I, Clancy JP, Nichols DP, Nick JA, De Boeck K, Solomon GM, Mall MA, Bolognese J, Bouisset F, den Hollander W, Paquette-Lamontagne N, Tomkinson N, Henig N, Elborn JS, Rowe SM. Sermet-Gaudelus I, et al. Among authors: de boeck k. J Cyst Fibros. 2019 Jul;18(4):536-542. doi: 10.1016/j.jcf.2018.10.015. Epub 2018 Nov 19. J Cyst Fibros. 2019. PMID: 30467074 Free PMC article.
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: de boeck k. 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.
Long-term safety and efficacy of tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years or older who are homozygous or heterozygous for Phe508del CFTR (EXTEND): an open-label extension study.
Flume PA, Biner RF, Downey DG, Brown C, Jain M, Fischer R, De Boeck K, Sawicki GS, Chang P, Paz-Diaz H, Rubin JL, Yang Y, Hu X, Pasta DJ, Millar SJ, Campbell D, Wang X, Ahluwalia N, Owen CA, Wainwright CE; VX14-661-110 study group. Flume PA, et al. Among authors: de boeck k. Lancet Respir Med. 2021 Jul;9(7):733-746. doi: 10.1016/S2213-2600(20)30510-5. Epub 2021 Feb 10. Lancet Respir Med. 2021. PMID: 33581080
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.
Wainwright CE, Elborn JS, Ramsey BW, Marigowda G, Huang X, Cipolli M, Colombo C, Davies JC, De Boeck K, Flume PA, Konstan MW, McColley SA, McCoy K, McKone EF, Munck A, Ratjen F, Rowe SM, Waltz D, Boyle MP; TRAFFIC Study Group; TRANSPORT Study Group. Wainwright CE, et al. Among authors: de boeck k. N Engl J Med. 2015 Jul 16;373(3):220-31. doi: 10.1056/NEJMoa1409547. Epub 2015 May 17. N Engl J Med. 2015. PMID: 25981758 Free PMC article. Clinical Trial.
276 results