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Article

Cystic fibrosis transmembrane conductance regulator modulators for cystic fibrosis: a new dawn?

Archives of disease in childhood - 1 Oct 2021

Edmondson Claire, Course Christopher William, Doull Iolo

Abstract excerpt

Cystic fibrosis (CF) is the most common life-limiting inherited condition in Caucasians. It is a multisystem autosomal recessive disorder caused by variants in the gene for cystic fibrosis transmembrane conductance regulator (CFTR) protein, a cell-surface localised chloride channel that regulates absorption and secretion of salt and water across epithelia. Until recently, the treatment for CF was predicated on...

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Identifiers

PMID: 33785533
DOI: 10.1136/archdischild-2020-320680

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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