Triple Therapy for Cystic Fibrosis <i>Phe508del</i> –Gating and –Residual Function Genotypes

Abstract

Elexacaftor-tezacaftor-ivacaftor was efficacious and safe in patients with <i>Phe508del</i>-gating or <i>Phe508del</i>-residual function genotypes and conferred additional benefit relative to previous CFTR modulators. (Funded by Vertex Pharmaceuticals; VX18-445-104 ClinicalTrials.gov number, NCT04058353.).