Ataluren

How it works

It promotes readthrough of premature stop codons during translation, allowing production of functional full-length dystrophin protein.

Prescribing in practice

• It is only appropriate where a nonsense mutation in the dystrophin gene has been confirmed by genetic testing, and is not effective for other mutation types.
• It should not be co-administered with intravenous aminoglycosides, which may increase the risk of nephrotoxicity, and renal function should be watched.
• Treatment must be initiated and supervised by a specialist in the management of Duchenne muscular dystrophy.

Monitoring

Monitor renal function and lipids periodically and assess ambulatory function to gauge ongoing clinical benefit.

Counselling the patient

• Take the medicine with food at the times instructed for steady absorption.
• Attend appointments for blood tests checking kidney function.
• Tell the team about all other medicines, especially any intravenous antibiotics.

Evidence & guidelines

Availability has been through managed access arrangements informed by clinical trials in nonsense-mutation Duchenne muscular dystrophy.