November 3, 2017Open Access

Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis

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Abstract

CFTR modulator therapy with tezacaftor-ivacaftor or ivacaftor alone was efficacious in patients with cystic fibrosis who were heterozygous for the Phe508del deletion and a CFTR residual-function mutation. (Funded by Vertex Pharmaceuticals and others; EXPAND ClinicalTrials.gov number, NCT02392234 .).

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Rowe et al. (Fri,) studied this question.

synapsesocial.com/papers/69edb3977beaa90d72fab925 https://doi.org/https://doi.org/10.1056/nejmoa1709847

Also Consider

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1Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508delCFTR2015 · 1,622 citations
2Cystic Fibrosis2006 · 4,415 citations
3Mutations in the Cystic Fibrosis Transmembrane Regulator Gene and In Vivo Transepithelial Potentials2006 · 179 citations
4Effect of genotype on phenotype and mortality in cystic fibrosis: a retrospective cohort study2003 · 391 citations
5Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del / F508del-CFTR or F508del / G551D-CFTR2017 · 178 citations

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