ataluren · Medicine question

Which patients is Translarna (ataluren) indicated to treat?

iatroX medicinesPublished 8 November 2026

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Translarna (ataluren) is indicated for ambulatory patients aged 2 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, confirmed by genetic testing.

Sources for this answer

Translarna 1000 mg granules for oral suspensionPTC Therapeutics Ltd · emc · SmPC §4.1 Therapeutic Indications · Version 14Document updated 31 March 2026

Sources checked 8 November 2026

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What driving precautions apply to patients who experience dizziness while taking ataluren?What lipid monitoring is recommended for patients with nonsense mutation Duchenne muscular dystrophy receiving ataluren?What was the most common adverse reaction to ataluren in placebo-controlled studies in patients with nonsense mutation Duchenne muscular dystrophy?How does ataluren affect exposure to adefovir?How does ataluren enable the production of full-length proteins despite a premature stop codon?

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