About miglustat
Glucosylceramide synthase inhibitor
Used for
- Treats mild-to-moderate type 1 Gaucher disease when enzyme replacement therapy is unsuitable.
- Treats Niemann-Pick disease type C neurological symptoms in adults and children.
Usual adult dose
Usually 100 mg three times daily for type 1 Gaucher disease or Niemann-Pick type C; dose may need adjustment for kidney problems.
Usual dose in children
For Niemann-Pick type C: under 12 years, dose is based on body surface area; ages 12–17, usually 200 mg three times daily.
Common side effects
- Diarrhea, gas, or abdominal discomfort
- Weight loss or reduced appetite
- Tremor
- Low platelet count
Important warnings
- Diarrhea may improve with dietary changes or prescribed treatment.
- May cause tremor or worsen existing neurological symptoms.
- Regular blood counts and neurological monitoring may be needed.
- Dose adjustment is required with impaired kidney function.
Pregnancy
Limited information; use during pregnancy only when clearly needed. Effective contraception is generally recommended during treatment.
Breastfeeding
It is not known whether miglustat passes into breast milk; breastfeeding is generally avoided during treatment.
Interactions
- Miglustat can reduce absorption of some medicines taken by mouth; check with a pharmacist.
- Imiglucerase may reduce miglustat exposure when used together.
AI-generated from general medical knowledge, not from this product's official leaflet. It may be incomplete or out of date. Not medical advice — ask a pharmacist or doctor before making any decision.