Safety and efficacy of elexacaftor/tezacaftor/ivacaftor in adolescents and adults with cystic fibrosis and F508del-gating and F508del-residual function genotypes: Results from an open-label extension study

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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

★ 3 Recommended by Dr Martin Frey, Faculty Member

★ 1 Recommended by Dr Marcus Hesse, Faculty Member

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

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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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★ 3 Recommended by Dr Martin Frey, Faculty Member

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Sermet-Gaudelus I, Letierce A, Berteloot L, Bonnel AS, Chen Y, Makani P, Kelly-Aubert M, Kanoun F, Penalva L, Bouleghem N, Bihouee T, Bui S, Corvol H, Houdouin V, Mittaine M, Tatopoulos A, Weiss L, Wizla N, Kapel N, Bessou A, Tiddens HAWM, Caudri D, Reix P, Marguet C, MODUL-CF study group

doi: 10.1016/S2213-2600(25)00248-6

Pulmonary HypertensionFurther Publications

Assessment of efficacy and tolerability of elexacaftor-tezacaftor-ivacaftor in an observational cohort study of "aged" people with cystic fibrosis

Ther Adv Respir Dis

Blankenship S, Flueckiger BV, Caleb G, Bailey J, Baker E, Garcia B, Krick S

doi: 10.1177/17534666251410246

Further Publications

Sweat chloride and lung function responses to elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis with two versus one responsive CFTR variants: an analysis of two real-world observational studies

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Burgel PR, Da Silva J, Girodon E, Durieu I, Reynaud-Gaubert M, Murris-Espin M, Chiron R, Grenet D, Ramel S, Mely L, Hamidfar R, Douvry B, Houdouin V, Audousset C, Macey J, Mittaine M, Weiss L, Cosson L, Danner-Boucher I, Reix P, Baravalle M, Burnet E, Sermet-Gaudelus I, Martin C, Paillasseur JL, French Cystic Fibrosis Reference Network study group

doi: 10.1016/S2213-2600(25)00190-0

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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Impact of Elexacaftor-Tezacaftor-Ivacaftor on fungal colonisation in children and adolescents with cystic fibrosis

★ 2 Recommended by Dr Martin Frey, Faculty Member

★ 1 Recommended by Dr Marcus Hesse, Faculty Member

J Cyst Fibros

Sitterlé E, Bonnel AS, Roussey-Bihouee T, Blondé A, Ribault M, Wizla N, Bresson C, Persat F, Maubon D, Leroy J, Hassani F, Benhala L, Mounier M, Znaidi S, Ferroni A, Bougnoux ME, Sermet-Gaudelus I, Mycologist and Modul-CF groups

doi: 10.1016/j.jcf.2026.02.012

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★ 1 Recommended by Dr Martin Frey, Faculty Member

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Alzaid M, Alshahrani T, Alotaibi R, Mukhtar G, Alanazi A, Alsadoon H, Eltahir S, Aldraihem A, Alotaibi W

doi: 10.1016/j.rmcr.2025.102264

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Severe hepatotoxicity is uncommon following the introduction of Elexacaftor/Tezacaftor/Ivacaftor: A real-world two-years follow-up study of the Danish cystic fibrosis cohort

★ 1 Recommended by Dr Martin Frey, Faculty Member

J Cyst Fibros

Højte C, Olsen MF, Faurholt-Jepsen D, Jørgensen MH, Pressler T, Leo-Hansen C, Bryrup T, Jeppesen M, Olesen HV, Petersen J, Katzenstein TL, Nielsen BU, Skov M, TransformCF Study Group

doi: 10.1016/j.jcf.2025.08.008

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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

Real-World Effectiveness and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Patients with Phe508del -Gating and -Residual Function Genotypes

Effect of elexacaftor-tezacaftor-ivacaftor on bronchial dilatations in adolescents with cystic fibrosis: a multicentre prospective observational study

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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

Impact of Elexacaftor-Tezacaftor-Ivacaftor on fungal colonisation in children and adolescents with cystic fibrosis

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