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Article

Ataluren: first global approval.

Drugs - 1 Sept 2014

Ryan Nicola J

Abstract excerpt

Nonsense mutations are implicated in 5-70 % of individual cases of most inherited diseases, including Duchenne muscular dystrophy (DMD) and cystic fibrosis. Ataluren (Translarna™) is an orally available, small molecule compound that targets nonsense mutations, and is the first drug in its class. Ataluren appears to allow cellular machinery to read through premature stop codons in mRNA, enabling the translation...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

Identifiers

PMID: 25193627
DOI: 10.1007/s40265-014-0287-4

Genes

DMD

Provenance

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.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

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Ataluren: first global approval.PMID 25193627Read ↗

Phase 2a Study of Ataluren-Mediated Dystrophin Production in Patients with Nonsense Mutation Duchenne Muscular DystrophySemantic proximity Read ↗

Ataluren treatment of patients with nonsense mutation dystrophinopathySemantic proximity Read ↗

European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin geneSemantic proximity Read ↗

Ataluren as an Agent for Therapeutic Nonsense SuppressionSemantic proximity Read ↗

Early treatment with Ataluren of a 2-year-old boy with nonsense mutation Duchenne dystrophy.Semantic proximity Read ↗

Opinion of the Italian Association of Myology on Ataluren for the Treatment of Nonsense Mutation Duchenne Muscular DystrophySemantic proximity Read ↗

Ataluren improves myelopoiesis and neutrophil chemotaxis by restoring ribosome biogenesis and reducing p53 levels in Shwachman–Diamond syndrome cellsSemantic proximity Read ↗

Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE RegistrySemantic proximity Read ↗

Neurology Care, Diagnostics, and Emerging Therapies of the Patient With Duchenne Muscular DystrophySemantic proximity Read ↗

Ataluren in cystic fibrosis: development, clinical studies and where are we now?Semantic proximity Read ↗

An Overview of Recent Therapeutics Advances for Duchenne Muscular Dystrophy.Semantic proximity Read ↗

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

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