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Article

Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor Therapy in Three Subjects with the Cystic Fibrosis Genotype Phe508del/Unknown and Advanced Lung Disease.

Genes - 29 Jul 2021

Terlizzi Vito, Colangelo Carmela, Marsicovetere Giovanni, D'Andria Michele, Francalanci Michela, Innocenti Diletta, Masi Eleonora, Avarello Angelo, Taccetti Giovanni, Amato Felice, Comegna Marika, Castaldo Giuseppe, Salvatore Donatello

Abstract excerpt

We evaluated the effectiveness and safety of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) in three subjects carrying the Phe508del/unknown CFTR genotype. An ex vivo analysis on nasal epithelial cells (NEC) indicated a significant improvement of CFTR gating activity after the treatment. Three patients were enrolled in an ELX/TEZ/IVA managed-access program, including subjects with the highest percent predicted...

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Identifiers

PMID: 34440351
DOI: 10.3390/genes12081178
PMCID: PMC8391133

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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