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Article

Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic fibrosis.

The Cochrane database of systematic reviews - 3 Mar 2023

Aslam Aisha A, Sinha Ian P, Southern Kevin W

Abstract excerpt

BACKGROUND: Cystic fibrosis (CF) is a common, life-shortening, genetic disorder in populations of Northern European descent caused by the mutation of a single gene that codes for the production of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This protein coordinates the transport of salt (and bicarbonate) across cell surfaces, and the mutation most notably affects the airways. In the...

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Identifiers

PMID: 36866921
DOI: 10.1002/14651858.CD012040.pub3
PMCID: PMC9983356

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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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