Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del)

Author:

Heneghan Matthew1,Southern Kevin W1,Murphy Jared2,Sinha Ian P1,Nevitt Sarah J34

Affiliation:

1. Department of Women's and Children's Health; University of Liverpool; Liverpool UK

2. Leighton Hospital; Crewe UK

3. Department of Health Data Science; University of Liverpool; Liverpool UK

4. Centre for Reviews and Dissemination; University of York; York UK

Publisher

Wiley

Subject

Pharmacology (medical)

Reference314 articles.

1. A phase 3, randomized, double-blind, controlled study evaluating the efficacy and safety of VX-445 combination therapy in subjects with cystic fibrosis who are heterozygous for the F508del mutation and a gating or residual function mutation (F/G and F/RF genotypes) https://www.clinicaltrialsregister.eu/ctr-search/trial/2018-002835-76/results

2. Triple therapy for cystic fibrosis Phe508del-gating and - residual function genotypes;Barry;New England Journal of Medicine,2021

3. A phase 3 study of VX-445 combination therapy in cystic fibrosis (CF) subjects heterozygous for f508del and a gating or residual function mutation (F/G and F/RF genotypes) www.who.int/trialsearch/Trial2.aspx?TrialID=EUCTR2018-002835-76-IE

4. A phase 3 study of VX-445 combination therapy in cystic fibrosis (CF) subjects heterozygous for F508del and a gating or residual function mutation (F/G and F/RF genotypes) clinicaltrials.gov/ct2/show/NCT04058353

5. Lumacaftor, an investigational CFTR corrector, in combination with ivacaftor, a CFTR potentiator, in CF patients with the F508del-CFTR mutation: phase 2 interim analysis;Boyle;Journal of Cystic Fibrosis,2013

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