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Theratyping cystic fibrosis patients to guide elexacaftor/tezacaftor/ivacaftor out-of-label prescription.
Dreano E, Burgel PR, Hatton A, Bouazza N, Chevalier B, Macey J, Leroy S, Durieu I, Weiss L, Grenet D, Stremler N, Ohlmann C, Reix P, Porzio M, Roux Claude P, Rémus N, Douvry B, Montcouquiol S, Cosson L, Mankikian J, Languepin J, Houdouin V, Le Clainche L, Guillaumot A, Pouradier D, Tissot A, Priou P, Mély L, Chedevergne F, Lebourgeois M, Lebihan J, Martin C, Zavala F, Da Silva J, Lemonnier L, Kelly-Aubert M, Golec A, Foucaud P, Marguet C, Edelman A, Hinzpeter A, de Carli P, Girodon E, Sermet-Gaudelus I, Pranke I; French CF Reference Network study group. Dreano E, et al. Among authors: golec a. Eur Respir J. 2023 Oct 19;62(4):2300110. doi: 10.1183/13993003.00110-2023. Print 2023 Oct. Eur Respir J. 2023. PMID: 37696564
Modulators of CFTR. Updates on clinical development and future directions.
Bardin E, Pastor A, Semeraro M, Golec A, Hayes K, Chevalier B, Berhal F, Prestat G, Hinzpeter A, Gravier-Pelletier C, Pranke I, Sermet-Gaudelus I. Bardin E, et al. Among authors: golec a. Eur J Med Chem. 2021 Mar 5;213:113195. doi: 10.1016/j.ejmech.2021.113195. Epub 2021 Jan 16. Eur J Med Chem. 2021. PMID: 33524685 Review.
Nonsense mutations accelerate lung disease and decrease survival of cystic fibrosis children.
Orenti A, Pranke I, Faucon C, Varilh J, Hatton A, Golec A, Dehillotte C, Durieu I, Reix P, Burgel PR, Grenet D, Tasset C, Gachelin E, Perisson C, Lepissier A, Dreano E, Tondelier D, Chevalier B, Weiss L, Kiefer S, Laurans M, Chiron R, Lemonnier L, Marguet C, Jung A, Edelman A, Kerem BS, Girodon E, Taulan-Cadars M, Hinzpeter A, Kerem E, Naehrlich L, Sermet-Gaudelus I; ECFSPR Steering group. Orenti A, et al. Among authors: golec a. J Cyst Fibros. 2023 Nov;22(6):1070-1079. doi: 10.1016/j.jcf.2023.06.005. Epub 2023 Jul 6. J Cyst Fibros. 2023. PMID: 37422433
Airway surface liquid acidification initiates host defense abnormalities in Cystic Fibrosis.
Simonin J, Bille E, Crambert G, Noel S, Dreano E, Edwards A, Hatton A, Pranke I, Villeret B, Cottart CH, Vrel JP, Urbach V, Baatallah N, Hinzpeter A, Golec A, Touqui L, Nassif X, Galietta LJV, Planelles G, Sallenave JM, Edelman A, Sermet-Gaudelus I. Simonin J, et al. Among authors: golec a. Sci Rep. 2019 Apr 24;9(1):6516. doi: 10.1038/s41598-019-42751-4. Sci Rep. 2019. PMID: 31019198 Free PMC article.
Author Correction: Airway surface liquid acidification initiates host defense abnormalities in Cystic Fibrosis.
Simonin J, Bille E, Crambert G, Noel S, Dreano E, Edwards A, Hatton A, Pranke I, Villeret B, Cottart CH, Vrel JP, Urbach V, Baatallah N, Hinzpeter A, Golec A, Touqui L, Nassif X, Galietta LJV, Planelles G, Sallenave JM, Edelman A, Sermet-Gaudelus I. Simonin J, et al. Among authors: golec a. Sci Rep. 2019 Nov 21;9(1):17535. doi: 10.1038/s41598-019-54253-4. Sci Rep. 2019. PMID: 31754179 Free PMC article.
Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10 kb C-to-T splicing mutation.
Oren YS, Irony-Tur Sinai M, Golec A, Barchad-Avitzur O, Mutyam V, Li Y, Hong J, Ozeri-Galai E, Hatton A, Leibson C, Carmel L, Reiter J, Sorscher EJ, Wilton SD, Kerem E, Rowe SM, Sermet-Gaudelus I, Kerem B. Oren YS, et al. Among authors: golec a. J Cyst Fibros. 2021 Sep;20(5):865-875. doi: 10.1016/j.jcf.2021.06.003. Epub 2021 Jul 2. J Cyst Fibros. 2021. PMID: 34226157 Free PMC article.
23 results