Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

Abstract

Elexacaftor-tezacaftor-ivacaftor was efficacious in patients with cystic fibrosis with Phe508del-minimal function genotypes, in whom previous CFTR modulator regimens were ineffective. (Funded by Vertex Pharmaceuticals; VX17-445-102 ClinicalTrials.gov number, NCT03525444.).

Keywords

IvacaftorCystic fibrosisMedicineCystic fibrosis transmembrane conductance regulatorPlaceboInternal medicinePathology

MeSH Terms

AdolescentAdultAminophenolsBenzodioxolesChildChloride Channel AgonistsChloridesCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorDouble-Blind MethodDrug CombinationsFemaleForced Expiratory VolumeGenotypeHumansIndolesMaleMutationPyrazolesPyridinesPyrrolidinesQuinolonesSweatYoung Adult

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Publication Info

Year: 2019
Type: article
Volume: 381
Issue: 19
Pages: 1809-1819
Citations: 1929
Access: Closed

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APA Style

                            
                                
                                    Peter G. Middleton, 
                                
                                    Marcus Mall, 
                                
                                    Pavel Dřevı́nek
                                
                                et al.
                            
                            (2019). 
                            Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele. 
                            New England Journal of Medicine
                            , 381
                            (19)
                            , 1809-1819.
                            https://doi.org/10.1056/nejmoa1908639
                        

Identifiers

DOI: 10.1056/nejmoa1908639
PMID: 31697873
PMCID: PMC7282384

Data Quality

Data completeness: 90%