Miglustat (By mouth)

mi-GLOO-stat

Treats type I Gaucher disease.

Drug classes

Endocrine-Metabolic Agent (About this – PubMed Health)

Uses

Uses of This Medicine

Miglustat is used to treat mild to moderate type 1 Gaucher disease in people who cannot be treated with an enzyme replacement therapy. Type 1 Gaucher disease is a disease you get from both your parents. People with type 1 Gaucher disease lack an enzyme (naturally occurring substance in your body) that breaks down a chemical in your body called glucosylceramide. Too much glucosylceramide causes liver and spleen enlargement, changes in the blood, and bone disease. Miglustat works by stopping the body from making glucosylceramide.

This medicine is available only with your doctor’s prescription.

Other uses (PubMed Health)

How To Use

Capsule

Take your medicine as directed. Your dose may need to be changed several times to find what works best for you.

Take this medicine at the same time each day.

Read and follow the patient instructions that come with this medicine. Talk to your doctor or pharmacist if you have any questions.

Missed dose: If you miss a dose of this medicine, skip the missed dose and go back to your regular dosing schedule. Do not double doses.

Store the medicine in a closed container at room temperature, away from heat, moisture, and direct light.

Drugs and Foods to Avoid

Ask your doctor or pharmacist before using any other medicine, including over-the-counter medicines, vitamins, and herbal products.

Some medicines can affect how miglustat works. Tell your doctor if you are using imiglucerase.

When Not To Use

This medicine is generally considered safe for most people. Talk to your doctor if you have concerns.

Warnings

Tell your doctor if you are pregnant or breastfeeding, or if you have kidney disease, tremors, or a low number of platelets in your blood.

This medicine may cause the following problems:

Peripheral neuropathy (a nerve problem) Tremors

Diarrhea is a common side effect of this medicine. Tell your doctor if you have diarrhea. He may suggest changes to your diet, such as eating fewer high-carbohydrate foods.

Your doctor will do lab tests at regular visits to check on the effects of this medicine. Keep all appointments.

Keep all medicine out of the reach of children. Never share your medicine with anyone.

Possible side effects

Summary More details

Call your doctor right away if you notice any of these side effects:

Allergic reaction: Itching or hives, swelling in your face or hands, swelling or tingling in your mouth or throat, chest tightness, trouble breathing

Numbness, tingling, or burning pain in your hands, arms, legs, or feet

Severe diarrhea, nausea, or vomiting

Trembling or shaking of the hands (either new or worse than usual)

Unusual bleeding, bruising, or weakness

If you notice these less serious side effects, talk with your doctor:

Headache or dizziness

Mild nausea, vomiting, constipation, gas, stomach pain or upset

Muscle cramps

Weight loss or loss of appetite

If you notice other side effects that you think are caused by this medicine, tell your doctor.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

More side effects of this drug

Brand names include

Zavesca

There may be other brand names for this medicine.