Miglustat is used to treat Gaucher disease type 1 (a condition in which a certain fatty substance is not broken down normally in the body and instead builds up in some organs and causes liver, spleen, bone, and blood problems). Miglustat is in a class of medications called enzyme inhibitors. It works by preventing the body from producing the fatty substance so that less of it will build up in the body and cause symptoms.

Side Effects Of Miglustat

Miglustat may cause side effects. Tell your doctor if any of these symptoms are severe or do not go away:

  • diarrhea
  • stomach pain or bloating
  • gas
  • loss of appetite
  • weight loss
  • upset stomach
  • vomiting
  • constipation
  • indigestion
  • dry mouth
  • weakness
  • muscle cramps, especially in the legs
  • the feeling of heaviness in the arms or legs
  • unsteadiness when walking
  • back pain
  • dizziness
  • nervousness
  • headache
  • memory problems
  • difficult or irregular menstruation (period)

Some side effects can be serious. The following symptoms are uncommon, but if you experience any of them, call your doctor immediately:

  • pain, burning, numbness, or tingling in your hands, arms, legs, or feet
  • shaking hands that you cannot control
  • changes in vision
  • easy bruising or bleeding
  • Miglustat may cause other side effects. Call your doctor if you have any unusual problems while taking this medication.

Warnings & Precautions

Before taking miglustat:

  • tell your doctor and pharmacist if you are allergic to miglustat, any other medications, or soy products.
  • tell your doctor and pharmacist what prescription and nonprescription medications, vitamins, nutritional supplements, and herbal products you are taking. Be sure to mention imiglucerase (Cerezyme). Your doctor may need to change the doses of your medications or monitor you carefully for side effects.
  • tell your doctor if you have or have ever had a tremor (shaking of your hands that you cannot control); pain, burning, tingling, or numbness in your hands or feet; any disease that affects your nervous system; or kidney disease.
  • tell your doctor if you are pregnant, plan to become pregnant, or are breast-feeding. You should use effective birth control to prevent pregnancy while you are taking miglustat. If you become pregnant while taking miglustat, call your doctor immediately. Miglustat may harm the fetus.
  • you should know that miglustat may damage sperm. Men who are taking miglustat should use effective birth control during treatment and for 3 months afterward.

Miglustat Dosage

Miglustat comes as a capsule to take by mouth. It is usually taken with or without food and with plenty of water up to three times a day. To help you remember to take miglustat, take it at around the same time(s) every day. Follow the directions on your prescription label carefully, and ask your doctor or pharmacist to explain any part you do not understand. Take miglustat exactly as directed. Do not take more or less of it or take it more often than prescribed by your doctor.

Swallow the capsules whole; do not split, chew, or crush them.

Miglustat controls Gaucher disease but does not cure it. Continue to take miglustat even if you feel well. Do not stop taking miglustat without talking to your doctor.


Keep all appointments with your doctor.

Do not let anyone else take your medication. Ask your pharmacist any questions you have about refilling your prescription.

It is important for you to keep a written list of all of the prescription and nonprescription (over-the-counter) medicines you are taking, as well as any products such as vitamins, minerals, or other dietary supplements. You should bring this list with you each time you visit a doctor or if you are admitted to a hospital. It is also important information to carry with you in case of emergencies.


All information has been provided courtesy of MedLinePlus from the National Library of Medicine and from the FDA.