The French compassionate programme of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with advanced lung disease and no F508del CFTR variant.
Burgel PR, Sermet-Gaudelus I, Durieu I, Kanaan R, Macey J, Grenet D, Porzio M, Coolen-Allou N, Chiron R, Marguet C, Douvry B, Dufeu N, Danner-Boucher I, Foucaud P, Lemonnier L, Girodon E, Da Silva J, Martin C; French CF Reference Network study group; Andrejak C, Priou P, Richaud-Thiriez B, Macey J, Montcouquiol S, Douvry B, Remus N, Fanton A, Coolen-Allou N, Gachelin E, Vuillard C, Audousset C, Duthoit L, Bellet-Fraysse E, Languepin J, Durieu I, Nove-Josserand R, Ohlmann C, Raynaud Q, Dufeu N, Chiron R, Billon Y, Danner-Boucher I, Tissot A, Leroy S, Aubourg F, Burnet E, Burgel PR, Carlier N, Da Silva J, Fajac I, Girodon E, Kanaan R, Honoré I, Martin C, Le Bourgeois M, Sermet-Gaudelus I, Le Clainche-Viala L, Corvol H, Dehillotte C, Foucaud P, Lemonnier L, Brinchault G, Ravoninjatovo B, Le Bihan J, Ramel S, Marguet C, Porzio M, Weiss L, Grenet D, de Miranda S, Cosson L, Mankikian J, Pouradier D.
Eur Respir J. 2023 May 5;61(5):2202437. doi: 10.1183/13993003.02437-2022. Print 2023 May.
PMID:36796836
Vanzacaftor-tezacaftor-deutivacaftor for children aged 6-11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial.
Hoppe JE, Kasi AS, Pittman JE, Jensen R, Thia LP, Robinson P, Tirakitsoontorn P, Ramsey B, Mall MA, Taylor-Cousar JL, McKone EF, Tullis E, Salinas DB, Zhu J, Chen YC, Rodriguez-Romero V, Sosnay PR, Davies G; VX21-121-105 Study Group.