What it is used for
- Historical use for ambulatory Duchenne muscular dystrophy confirmed to result from a nonsense mutation in the dystrophin gene
- Potential use only where it remains authorised, under a specialist centre and after confirmatory genetic testing
How it is used
- Do not start or renew the medicine until the neuromuscular centre confirms its current authorisation and relevance to your case
- Prepare the granules in a leaflet-permitted liquid or soft food and take the complete mixture without storing leftovers
- Use the correct sachet contents as prescribed and do not exchange sachets or alter the dose yourself
- Attend mobility, kidney, liver, lipid and blood-pressure monitoring set by the centre
- If a dose is missed or vomiting occurs, follow the leaflet or contact the centre and do not double it
Possible side effects
- Nausea, vomiting or abdominal pain
- Headache
- Diarrhoea or wind
- Reduced appetite or weight loss
- Changed kidney or lipid tests
Who should not use it
- Allergy to ataluren or another ingredient
- Absence of a confirmed nonsense mutation in the dystrophin gene
- Use of an intravenous aminoglycoside where the label prohibits the combination
- Starting treatment in a country where it has no valid authorisation
Before and while using it
- Clinical benefit was not confirmed in required studies, so uncertainty must be explained to the patient and family
- Kidney disease, dehydration or a kidney-toxic medicine can increase risk and requires urgent review
- Reduced urine, swelling or profound tiredness may indicate kidney injury
- Lipids or blood pressure can rise, particularly with corticosteroids, and require centre monitoring
- Do not use it for another mutation or a muscular dystrophy other than Duchenne because the mechanism does not apply
- Stopping or continuing after a change in walking ability is a specialist decision, not a patient-led one
Medicine interactions
- Intravenous aminoglycosides can increase kidney toxicity and the combination may be prohibited
- Rifampicin and inducers of selected conjugating enzymes can reduce exposure
- Medicines using kidney transporters or capable of kidney injury require review
- Corticosteroids are part of many patients' plans and have their own risks and monitoring
Pregnancy and breastfeeding
- Tell the centre if you are pregnant or planning pregnancy; human information is limited and authorisation and need must be checked
- Do not breastfeed during treatment where the local leaflet prohibits it
How to store it
- Keep sachets dry in their original pack, protected from heat and out of children's reach, and use prepared mixture as the leaflet directs
Get medical help straight away if
- Your face, lips or tongue swell, or you find it hard to breathe.
- You get a severe rash, blistering or peeling skin.
- A side effect worries you or doesn’t go away: contact your doctor or pharmacist.
Questions about this medicine?
Talk to a doctor by video, or send your question by text.
Source: Standard international medicine references
This guide is general information and doesn’t replace advice from your doctor or pharmacist. Don’t start, stop or change a medicine without asking them, and always read the leaflet in the pack.