What it is used for
- Long-term treatment of confirmed Fabry disease to replace the missing enzyme
- Slowing the build-up of the fatty substance and damage to the kidneys and heart, and easing nerve pain in some patients
How it is used
- It is given as a drip into a vein by a nurse or doctor, and may be given at home after training if your specialist decides
- It is given at regular intervals set by your specialist, and treatment is usually lifelong
- You may be given medicines before the drip to prevent infusion reactions
- Attend regular kidney, heart and nerve check-ups
Possible side effects
- Infusion reactions such as chills, fever, flushing, headache and nausea
- Tiredness or pain in the arms and legs
- Dizziness or palpitations
- Rarely, a severe allergic reaction
Who should not use it
- Severe allergy to the medicine or any of its ingredients
Before and while using it
- Tell the nurse at once if you feel breathless or get a rash or facial swelling during or after the drip
- The body can make antibodies to the medicine that increase infusion reactions or reduce its effect
- People with severe heart disease need close monitoring during the drip
- Do not switch to another enzyme product unless your specialist decides
Medicine interactions
- Chloroquine, amiodarone and gentamicin may reduce the enzyme's activity and are not recommended with it; 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 or as your care team instructs
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.