Last reviewed 2026-04-20 · How we verify

Translarna (ATALUREN)

Translarna (ataluren) is a small molecule modality developed by PTC Therapeutics International Limited, targeting flavin reductase (NADPH) to treat Duchenne muscular dystrophy. It is currently owned by PTC Therapeutics International Limited and has been approved for its indication. The commercial status of Translarna is patented, and its safety considerations include potential liver enzyme elevations and gastrointestinal side effects. Translarna works by bypassing premature stop codons in the genetic code, allowing for the production of functional dystrophin protein. Further clinical data and long-term studies are needed to fully understand its efficacy and safety profile.

At a glance

Approved indications

• Duchenne muscular dystrophy

Common side effects

No common side effects on file.

Key clinical trials

• Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy (PHASE3)
• Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD) (PHASE3)
• Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
• A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) (PHASE2)
• Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome (PHASE2)
• Pembrolizumab With Ataluren in Patients With Metastatic pMMR and dMMR Colorectal Carcinoma or Metastatic dMMR Endometrial Carcinoma: the ATAPEMBRO Study (PHASE1,PHASE2)
• Study of Ataluren in Participants With Nonsense Mutation Aniridia (PHASE2)
• A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) (PHASE2)

Primary sources

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