Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del

First experience in Switzerland in Phe508del homozygous cystic fibrosis patients with end-stage pulmonary disease enrolled in a lumacaftor-ivacaftor therapy trial - preliminary results

Swiss Med Wkly.

Murer C, Huber LC, Kurowski T, Hirt A, Robinson CA, Bürgi U, Benden C

doi: 10.4414/smw.2018.14593.

Pulmonary Fibrosis

Effects of Lumacaftor/Ivacaftor Therapy on CFTR Function in Phe508del Homozygous Patients with Cystic Fibrosis

Am J Respir Crit Care Med.

Graeber SY, Dopfer C, Naehrlich L, Gyulumyan L, Scheuermann H, Hirtz S, Wege S, Mairbäurl H, Dorda M, Hyde R, Bagheri-Hanson A, Rueckes-Nilges C, Fischer S, Mall MA, Tümmler B

doi: 10.1164/

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Further Publications

Long-term safety and efficacy of tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years or older who are homozygous or heterozygous for Phe508del CFTR (EXTEND): an open-label extension study

★ 2 Recommended by Dr Martin Frey, Faculty Member

★ 2 Recommended by Dr Marcus Hesse, Faculty Member

Lancet Respir Med

Flume PA, Biner RF, Downey DG, Brown C, Jain M, Fischer R, De Boeck K, Sawicki GS, Chang P, Paz-Diaz H, Rubin JL, Yang Y, Hu X, Pasta DJ, Millar SJ, Campbell D, Wang X, Ahluwalia N, Owen CA, Wainwright CE, VX14-661-110 study group.

doi: 10.1016/S2213-2600(20)30510-5

Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled phase 3 trial

Lancet Respir Med.

Ratjen F, Hug C, Marigowda G, Tian S, Huang X, Stanojevic S, Milla CE, Robinson PD, Waltz D, Davies JC; VX14-809-109 investigator group

doi: 10.1016/

Pulmonary Fibrosis

Tezacaftor-Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis

N Engl J Med.

Rowe SM, Daines C, Ringshausen FC, Kerem E, Wilson J, Tullis E, Nair N, Simard C, Han L, Ingenito EP, McKee C, Lekstrom-Himes J, Davies JC

doi: 10.1056/

RECOMMENDED

Pulmonary Fibrosis

Rapid Improvement After Starting Elexacaftor-tezacaftor-ivacaftor in Patients with Cystic Fibrosis and Advanced Pulmonary Disease

★ 3 Recommended by Dr Martin Frey, Faculty Member

★ 1 Recommended by Dr Marcus Hesse, Faculty Member

Am J Respir Crit Care Med

Burgel PR, Durieu I, Chiron R, Ramel S, Danner-Boucher I, Prevotat A, Grenet D, Marguet C, Reynaud-Gaubert M, Macey J, Mely L, Fanton A, Quetant S, Lemonnier L, Paillasseur JL, Da Silva J, Martin C, French Cystic Fibrosis Reference Network study group.

doi: 10.1164/rccm.202011-4153OC

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Pulmonary Fibrosis

A phase 3, double-blind, parallel-group study to evaluate the efficacy and safety of tezacaftor in combination with ivacaftor in participants 6 through 11 years of age with cystic fibrosis homozygous for F508del or heterozygous for the F508del-CFTR mutation and a residual function mutation

★ 2 Recommended by Dr Martin Frey, Faculty Member

J Cyst Fibros.

Davies JC, Sermet-Gaudelus I, Naehrlich L, Harris RS, Campbell D, Ahluwalia N, Short C, Haseltine E, Panorchan P, Saunders C, Owen CA, Wainwright CE, VX16-661-115 Investigator Group.

doi: 10.1016/j.jcf.2020.07.023.

RECOMMENDED

Pulmonary Fibrosis

Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial

★ 3 Recommended by Dr Martin Frey, Faculty Member

Lancet.

Heijerman HGM, McKone EF, Downey DG, Van Braeckel E, Rowe SM, Tullis E, Mall MA, Welter JJ, Ramsey BW, McKee CM, Marigowda G, Moskowitz SM, Waltz D, Sosnay PR, Simard C, Ahluwalia N, Xuan F, Zhang Y, Taylor-Cousar JL, McCoy KS, VX17-445-103 Trial Group.

doi: 10.1016/S0140-6736(19)32597-8.

RECOMMENDED

Pulmonary Fibrosis

Safety, pharmacokinetics, and pharmacodynamics of lumacaftor and ivacaftor combination therapy in children aged 2-5 years with cystic fibrosis homozygous for F508del-CFTR: an open-label phase 3 study

Lancet Respir Med.

McNamara JJ, McColley SA, Marigowda G, Liu F, Tian S, Owen CA, Stiles D, Li C, Waltz D, Wang LT, Sawicki GS

doi: 10.1016/S2213-2600(18)30460-0.

Related content

First experience in Switzerland in Phe508del homozygous cystic fibrosis patients with end-stage pulmonary disease enrolled in a lumacaftor-ivacaftor therapy trial - preliminary results

Effects of Lumacaftor/Ivacaftor Therapy on CFTR Function in Phe508del Homozygous Patients with Cystic Fibrosis

Long-term safety and efficacy of tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years or older who are homozygous or heterozygous for Phe508del CFTR (EXTEND): an open-label extension study

Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled phase 3 trial

Tezacaftor-Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis

Rapid Improvement After Starting Elexacaftor-tezacaftor-ivacaftor in Patients with Cystic Fibrosis and Advanced Pulmonary Disease

A phase 3, double-blind, parallel-group study to evaluate the efficacy and safety of tezacaftor in combination with ivacaftor in participants 6 through 11 years of age with cystic fibrosis homozygous for F508del or heterozygous for the F508del-CFTR mutation and a residual function mutation

Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial

Safety, pharmacokinetics, and pharmacodynamics of lumacaftor and ivacaftor combination therapy in children aged 2-5 years with cystic fibrosis homozygous for F508del-CFTR: an open-label phase 3 study