What it is used for
- Long-term treatment of Pompe disease (acid alpha-glucosidase deficiency)
- Helping to preserve muscle strength and breathing function
How it is used
- It is given as a slow drip into a vein in a hospital or specialist centre; your doctor works out the dose from your weight
- You may be given medicines beforehand to reduce reactions, and you are watched during and after the infusion
- Treatment is long term; do not miss infusion appointments and follow your doctor's plan
Possible side effects
- Infusion reactions such as fever, chills, rash, itching or headache
- Nausea, tiredness or muscle pain
- Rarely, a severe allergic reaction with difficulty breathing, facial swelling or low blood pressure
Who should not use it
- It is not given to people who have had a life-threatening allergic reaction to it, unless the specialist decides otherwise
Before and while using it
- Tell the medical team straight away if you feel short of breath, swollen or dizzy during the infusion
- People with severe heart or breathing weakness are at higher risk of problems during the infusion
- The body can make antibodies against the medicine; your doctor may order tests to check for them
Medicine interactions
- Tell your doctor or pharmacist about all your medicines, including herbal products and supplements
Pregnancy and breastfeeding
- Tell your doctor if you are pregnant, planning a pregnancy or breastfeeding; the specialist decides about continuing treatment after weighing benefits and risks
How to store it
- The hospital or pharmacy stores it in the fridge as the maker directs; it must not be frozen.
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.