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Longer term follow-up of abdominal symptoms (CFAbd-Score) after initiation of Elexacaftor / Tezacaftor / Ivacaftor in adults with cystic fibrosis
Pulmonary Fibrosis
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Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Adults and Adolescents with Cystic Fibrosis and at Least One F508del Allele: A Phase 3, Open-Label Extension Study
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Am J Respir Crit Care Med
Daines CL, Polineni D, Tullis E, Costa S, Linnemann RW, Mall MA, McKone EF, Quon BS, Ringshausen FC, Selvadurai H, Taylor-Cousar JL, Withers NJ, Sawicki GS, Lee T, Ahluwalia N, Morlando Geiger J, Jennings M, Tan YV, Waltz D, Ramsey B, Griese M, VX17-445-105 Study Group
doi: 10.1164/rccm.202411-2231OC
Pulmonary Fibrosis
LONGITUDE: An observational study of the long-term effectiveness of elexacaftor/tezacaftor/ivacaftor in people aged ≥12 years with cystic fibrosis using data from the United Kingdom Cystic Fibrosis Registry - 2-year analysis
J Cyst Fibros
Vega-Hernandez G, MacGregor G, Wilfin A, Adams F, Haugh C, Baxter CA, Wöhling H, Clarke SL, Charman SC, Carr SB
doi: 10.1016/j.jcf.2025.04.012
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Pulmonary Fibrosis
The expanded French compassionate programme for elexacaftor-tezacaftor-ivacaftor use in people with cystic fibrosis without a F508del CFTR variant: a real-world study
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Lancet Respir Med
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
doi: 10.1016/S2213-2600(24)00208-X
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Vanzacaftor-tezacaftor-deutivacaftor versus elexacaftor-tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years and older (SKYLINE Trials VX20-121-102 and VX20-121-103): results from two randomised, active-controlled, phase 3 trials
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Lancet Respir Med
Keating C, Yonker LM, Vermeulen F, Prais D, Linnemann RW, Trimble A, Kotsimbos T, Mermis J, Braun AT, O'Carroll M, Sutharsan S, Ramsey B, Mall MA, Taylor-Cousar JL, McKone EF, Tullis E, Floreth T, Michelson P, Sosnay PR, Nair N, Zahigian R, Martin H, Ahluwalia N, Lam A, Horsley A
doi: 10.1016/S2213-2600(24)00411-9
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Pulmonary Fibrosis
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Thorax
Maher RE, Cytlak-Chaudhuri UM, Aleem S, Barry P, Brice DP, Caamaño Gutiérrez E, Driver K, Emmott E, Rothwell A, Smith E, Travis M, Lee D, McNamara PS, Waller I, Smith JA, Jones A, Lord RW
doi: 10.1136/thorax-2024-222242
Pulmonary FibrosisFurther Publications
Elexacaftor/tezacaftor/ivacaftor and inflammation in children and adolescents with cystic fibrosis: a retrospective dual-center cohort study
Ther Adv Respir Dis
Pepe A, Fevola C, Dolce D, Campana S, Ravenni N, Taccetti G, Salvatore D, Terlizzi V
doi: 10.1177/17534666251314706
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Real-World Effectiveness and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Patients with Phe508del -Gating and -Residual Function Genotypes
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Salvatore D, Iacotucci P, Pepe A, Terminiello A, Majo F, Carnovale V, Vitullo P, Messore B, Parisi GF, Calderazzo MA, Leonetti G, Terlizzi V
doi: 10.1016/j.rmed.2025.108221
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Impact of availability of a highly effective Cystic Fibrosis treatment (elexacaftor/tezacaftor/ivacaftor) on lung transplant waitlist and lung transplantation trends in the US
Respir Med
Merlo C, Geiger JM, Wang Z, Lyden GR, Schladt DP, McGarry L
doi: 10.1016/j.rmed.2025.108171
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Thorax
Merlo CA, Thorat T, DerSarkissian M, McGarry LJ, Nguyen C, Gu YM, Healy J, Rubin JL, Brookhart MA
doi: 10.1136/thorax-2023-220558