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

ABSTRACT:

SUBMITTER: Aslam AA

PROVIDER: S-EPMC9983356 | biostudies-literature | 2023 Mar

REPOSITORIES: biostudies-literature

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

Aslam Aisha A AA Sinha Ian P IP Southern Kevin W KW

The Cochrane database of systematic reviews 20230303

<h4>Background</h4>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 lungs of people with CF, the defective protein compromises mucociliary clearanc ...[more]

PMID: 36866921

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