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Article

Effect of Ataluren on dystrophin mutations.

Journal of cellular and molecular medicine - 1 Jun 2020

Berger Joachim, Li Mei, Berger Silke, Meilak Michelle, Rientjes Jeanette, Currie Peter D

Abstract excerpt

Duchenne muscular dystrophy is a severe muscle wasting disease caused by mutations in the dystrophin gene (dmd). Ataluren has been approved by the European Medicines Agency for treatment of Duchenne muscular dystrophy. Ataluren has been reported to promote ribosomal read-through of premature stop codons, leading to restoration of full-length dystrophin protein. However, the mechanism of Ataluren action has not...

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Identifiers

PMID: 32343037
DOI: 10.1111/jcmm.15319
PMCID: PMC7299694

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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