Ataluren is a medication used for the treatment of Duchenne muscular dystrophy. Ataluren is a novel, orally administered drug that targets nonsense mutations.
- Mechanism
- Curator reviewed
Ataluren enables ribosomal readthrough of mRNA containing premature stop codons that otherwise would result in premature termination of protein chains. Use of ataluren allows cellular machinery to bypass nonsense mutations in genetic material, continue the translation process, and thereby restore the production of a full-length, functional protein.
The research on the effects of Ataluren on the translation and stability of nonsense-containing mRNA in vitor show that Ataluren promoted readthrough at each of the nonsense codons, showing maximal activity with UGA, while having no effect on mRNA levels. Unlike the stable cell line assays, Ataluren did not discriminate significantly between the UAG and UAA mRNAs. Ataluren was a more potent nonsense-suppressing agent than gentamicin, and exhibited 4- to 15-fold stimulation of in vitro readthrough relative to the controls at levels similar to those in the stable cell reporter assays. These results indicate that Ataluren modulates termination efficiency at premature nonsense codons.
- Primary indication
- Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk.Curator reviewed · 1 structured indication
- Formula / weight
- C15H9FN2O3 · 284.242 g/mol (avg)
- Code names
- PTC-124
Resolves to
OOUGLTULBSNHNF-UHFFFAOYSA-NSMILESOC(=O)C1=CC=CC(=C1)C1=NOC(=N1)C1=CC=CC=C1FWhat you can answer from here — as of June 08, 2025
Which drugs share a target with Ataluren, and which of those have an active Phase 3 trial?
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