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Article

Real-world effectiveness and safety of elexacaftor/tezacaftor/ivacaftor in cystic fibrosis patients with Phe508del -gating and -residual function genotypes.

Respiratory medicine - 1 Jan 2025

Salvatore Donatello, Iacotucci Paola, Pepe Angela, Terminiello Alberto, Majo Fabio, Carnovale Vincenzo, Vitullo Pamela, Messore Barbara, Parisi Giuseppe Fabio, Calderazzo Maria Adelaide, Leonetti Giuseppina, Terlizzi Vito

Abstract excerpt

BACKGROUND: Cystic fibrosis (CF) is a genetic disease caused by CFTR protein dysfunction. Elexacaftor/tezacaftor/ivacaftor (ETI) shows promise in improving outcomes for people with CF (pwCF) with Phe508del combined with gating (F/G) or residual function (F/RF) mutations, though real-world evidence remains scarce. OBJECTIVES: This study evaluated the effectiveness and safety of ETI therapy in pwCF with F/G and...

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Identifiers

PMID: 40571167
DOI: 10.1016/j.rmed.2025.108221

Genes

CFTR

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

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Real-world effectiveness and safety of elexacaftor/tezacaftor/ivacaftor in cystic fibrosis patients with Phe508del -gating and -residual function genotypes.PMID 40571167Read ↗

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