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116 results

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Page 1
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: fajac i. 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.
Understanding and addressing the needs of people with cystic fibrosis in the era of CFTR modulator therapy.
Hisert KB, Birket SE, Clancy JP, Downey DG, Engelhardt JF, Fajac I, Gray RD, Lachowicz-Scroggins ME, Mayer-Hamblett N, Thibodeau P, Tuggle KL, Wainwright CE, De Boeck K. Hisert KB, et al. Among authors: fajac i. Lancet Respir Med. 2023 Oct;11(10):916-931. doi: 10.1016/S2213-2600(23)00324-7. Epub 2023 Sep 9. Lancet Respir Med. 2023. PMID: 37699420 Review.
Advancing the pipeline of cystic fibrosis clinical trials: a new roadmap with a global trial network perspective.
Mayer-Hamblett N, Clancy JP, Jain R, Donaldson SH, Fajac I, Goss CH, Polineni D, Ratjen F, Quon BS, Zemanick ET, Bell SC, Davies JC, Jain M, Konstan MW, Kerper NR, LaRosa T, Mall MA, McKone E, Pearson K, Pilewski JM, Quittell L, Rayment JH, Rowe SM, Taylor-Cousar JL, Retsch-Bogart G, Downey DG. Mayer-Hamblett N, et al. Among authors: fajac i. Lancet Respir Med. 2023 Oct;11(10):932-944. doi: 10.1016/S2213-2600(23)00297-7. Epub 2023 Sep 9. Lancet Respir Med. 2023. PMID: 37699421 Review.
ECFS standards of care on CFTR-related disorders: Towards a comprehensive program for affected individuals.
De Wachter E, De Boeck K, Sermet-Gaudelus I, Simmonds NJ, Munck A, Naehrlich L, Barben J, Boyd C, Veen SJ, Carr SB, Fajac I, Farrell PM, Girodon E, Gonska T, Grody WW, Jain M, Jung A, Kerem E, Raraigh KS, van Koningsbruggen-Rietschel S, Waller MD, Southern KW, Castellani C; ECFS Diagnostic Network Working Group. De Wachter E, et al. Among authors: fajac i. J Cyst Fibros. 2024 Feb 21:S1569-1993(24)00011-0. doi: 10.1016/j.jcf.2024.01.012. Online ahead of print. J Cyst Fibros. 2024. PMID: 38388234
Gathering real-world compassionate data to expand eligibility for elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with N1303K or other rare CFTR variants: a viewpoint.
Burgel PR, Sermet-Gaudelus I, Girodon E, Kanaan R, Le Bihan J, Remus N, Ravoninjatovo B, Grenet D, Porzio M, Houdouin V, Le Clainche-Viala L, Durieu I, Nove-Josserand R, Languepin J, Coltey B, Guillaumot A, Audousset C, Chiron R, Weiss L, Fajac I, Da Silva J, Martin C; French CF Reference Network study group. Burgel PR, et al. Among authors: fajac i. Eur Respir J. 2024 Jan 25;63(1):2301959. doi: 10.1183/13993003.01959-2023. Print 2024 Jan. Eur Respir J. 2024. PMID: 38242629 No abstract available.
Standards for the care of people with cystic fibrosis; establishing and maintaining health.
Southern KW, Addy C, Bell SC, Bevan A, Borawska U, Brown C, Burgel PR, Button B, Castellani C, Chansard A, Chilvers MA, Davies G, Davies JC, De Boeck K, Declercq D, Doumit M, Drevinek P, Fajac I, Gartner S, Georgiopoulos AM, Gursli S, Gramegna A, Hansen CM, Hug MJ, Lammertyn E, Landau EEC, Langley R, Mayer-Hamblett N, Middleton A, Middleton PG, Mielus M, Morrison L, Munck A, Plant B, Ploeger M, Bertrand DP, Pressler T, Quon BS, Radtke T, Saynor ZL, Shufer I, Smyth AR, Smith C, van Koningsbruggen-Rietschel S. Southern KW, et al. Among authors: fajac i. J Cyst Fibros. 2024 Jan;23(1):12-28. doi: 10.1016/j.jcf.2023.12.002. Epub 2023 Dec 21. J Cyst Fibros. 2024. PMID: 38129255 Free article. Review.
Standards for the care of people with cystic fibrosis (CF).
Southern KW, Burgel PR, Castellani C, De Boeck K, Davies JC, Dunlevy F, Fajac I, Gramegna A, Lammertyn E, Middleton PG, Ratjen F, van Koningsbruggen-Rietschel S. Southern KW, et al. Among authors: fajac i. J Cyst Fibros. 2023 Nov;22(6):961-962. doi: 10.1016/j.jcf.2023.09.009. Epub 2023 Oct 3. J Cyst Fibros. 2023. PMID: 37798158 No abstract available.
Cystic fibrosis.
Fajac I, Burgel PR. Fajac I, et al. Presse Med. 2023 Jul 27;52(3):104169. doi: 10.1016/j.lpm.2023.104169. Online ahead of print. Presse Med. 2023. PMID: 37516246
AQP5, a second gene at play with CFTR in aquagenic palmoplantar keratoderma.
Sperelakis-Beedham B, Lopez M, Bourrat E, Gaitch N, Houriez F, Martinez B, Fajac I, Burgel PR, Hickman G, Audrézet MP, Gonde D, Cabet F, Gerfaud-Valentin M, Nove-Josserand R, Raynal C, Pagin A, Reboul MP, de Becdelièvre A, Bienvenu T, Callebaut I, Girodon E. Sperelakis-Beedham B, et al. Among authors: fajac i. J Eur Acad Dermatol Venereol. 2023 May;37(5):e654-e657. doi: 10.1111/jdv.18869. Epub 2023 Jan 24. J Eur Acad Dermatol Venereol. 2023. PMID: 36645858 No abstract available.
116 results