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The expanded French compassionate programme for elexacaftor-tezacaftor-ivacaftor use in people with cystic fibrosis without a F508del CFTR variant: a real-world study.
Burgel PR, Sermet-Gaudelus I, Girodon E, Durieu I, Houdouin V, Audousset C, Macey J, Grenet D, Porzio M, Murris-Espin M, Reix P, Baravalle M, Belleguic C, Mely L, Verhille J, Weiss L, Reynaud-Gaubert M, Mittaine M, Hamidfar R, Ramel S, Cosson L, Douvry B, Danner-Boucher I, Foucaud P, Roy C, Burnet E, Raynal C, Audrezet MP, Da Silva J, Martin C; French Cystic Fibrosis Reference Network study group. Burgel PR, et al. Lancet Respir Med. 2024 Nov;12(11):888-900. doi: 10.1016/S2213-2600(24)00208-X. Epub 2024 Aug 13. Lancet Respir Med. 2024. PMID: 39151434
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: lebihan j. Eur Respir J. 2023 Oct 19;62(4):2300110. doi: 10.1183/13993003.00110-2023. Print 2023 Oct. Eur Respir J. 2023. PMID: 37696564 Free article.