Triple Therapy for Cystic Fibrosis Phe508del-Gating and -Residual Function Genotypes.

doi:10.1056/NEJMoa2100665

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Article

Triple Therapy for Cystic Fibrosis Phe508del-Gating and -Residual Function Genotypes.

The New England journal of medicine - 26 Aug 2021

Barry Peter J, Mall Marcus A, Álvarez Antonio, Colombo Carla, de Winter-de Groot Karin M, Fajac Isabelle, McBennett Kimberly A, McKone Edward F, Ramsey Bonnie W, Sutharsan Sivagurunathan, Taylor-Cousar Jennifer L, Tullis Elizabeth, Ahluwalia Neil, Jun Lucy S, Moskowitz Samuel M, Prieto-Centurion Valentin, Tian Simon, Waltz David, Xuan Fengjuan, Zhang Yaohua, Rowe Steven M, Polineni Deepika

Abstract excerpt

BACKGROUND: Elexacaftor-tezacaftor-ivacaftor is a small-molecule cystic fibrosis transmembrane conductance regulator (CFTR) modulator regimen shown to be efficacious in patients with at least one Phe508del allele, which indicates that this combination can modulate a single Phe508del allele. In patients whose other CFTR allele contains a gating or residual function mutation that is already effectively treated with...

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PMID: 34437784
DOI: 10.1056/NEJMoa2100665
PMCID: PMC8982185

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