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Article

Real-World Clinical Outcomes of Lumacaftor-Ivacaftor or Tezacaftor-Ivacaftor Therapy in Children With Cystic Fibrosis Homozygous for Phe508del.

Pediatric pulmonology - 1 Jun 2026

Aerssens Nele, Vermeulen François, Boon Mieke, Proesmans Marijke

Abstract excerpt

BACKGROUND: Cystic fibrosis (CF) is a progressive, life-shortening disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CFTR modulator therapies targeting the common Phe508del mutation have been introduced in clinical practice. This study evaluates the real-world effects of 1 year of dual CFTR modulator therapy - lumacaftor/ivacaftor or tezacaftor/ivacaftor - in...

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Identifiers

PMID: 42272190
DOI: 10.1002/ppul.71701

Genes

CFTR

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

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Real-World Clinical Outcomes of Lumacaftor-Ivacaftor or Tezacaftor-Ivacaftor Therapy in Children With Cystic Fibrosis Homozygous for Phe508del.PMID 42272190Read ↗

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