What it is used for
- Long-term treatment of confirmed Pompe disease in infants, children and adults
- Helping to improve or maintain muscle strength, breathing and heart function
How it is used
- It is given as a drip into a vein by a trained team with emergency equipment at hand
- It is given at regular intervals set by your specialist, and treatment is usually long term
- Medicines may be given before the drip, and you will be watched during and after it
Possible side effects
- Infusion reactions: rash, fever, flushing and a fast heartbeat
- Headache, nausea or vomiting
- Tiredness
- Rarely, a severe allergic reaction or immune reactions affecting the skin or kidneys
Who should not use it
- A previous life-threatening allergic reaction to it, unless a specialist decides to try again under close supervision
Before and while using it
- It can cause a severe allergic reaction; tell the team at once about breathlessness, facial swelling or a rash
- Infants with weak heart or breathing are at higher risk during the drip and need close monitoring
- The body can make antibodies that reduce how well it works; your doctor monitors this
- Tell the team if you are unwell or have a fever on the day of the drip
Medicine interactions
- No important drug interactions are known, but tell your doctor about all your medicines
Pregnancy and breastfeeding
- Information is limited; your specialist decides about continuing it in pregnancy or breastfeeding
How to store it
- Vials are kept in the fridge and prepared in hospital
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.