Indication :
- The drug contains the active ingredient miglustat, which belongs to the group of drugs that affect metabolism. It is used to treat two diseases:
- The drug is used to treat Gaucher disease type 1 of mild to moderate severity in adults.
- In type 1 Gaucher disease, the substance glucosylceramide is not excreted from the body. It accumulates in certain cells of the immune system. This can lead to an enlarged liver and spleen, blood changes and bone diseases.
- The usual treatment for type 1 Gaucher disease is enzyme replacement therapy. The drug is used only in patients for whom enzyme replacement therapy is not possible.
- The drug is also used in Niemann-Pick disease type C to treat progressive neurological symptoms in adults and children.
- If you have Niemann-Pick disease type C, fats such as: B. glycosphingolipids in your brain cells. This can lead to problems with neurological functions such as slowed eye movements, balance, swallowing and memory, as well as seizures.
- The principle of action is to inhibit the enzyme “glucosylceramide synthase”. It is responsible for the first stage of the synthesis of most glycosphingolipids.
- The drug is used to treat Gaucher disease type 1 of mild to moderate severity in adults.
Dosage :
- Always take this medicine exactly as your doctor told you. If you are not sure, ask your doctor or pharmacist.
- For Gaucher disease type 1: For adults, the usual dose is one capsule (100 mg) three times daily (morning, afternoon and evening). This means a maximum of three capsules per day (300 mg) .
- For Niemann-Pick disease type C: Adults and adolescents (over 12 years of age), the usual dose is two capsules (200 mg) three times daily (morning, afternoon and evening). This means a maximum six capsules per day (600 mg).
- In children under 12 years of age, the dose is adjusted by the doctor depending on Niemann-Pick disease type C.
- If you have kidney problems, you may be given a lower starting dose. If you have diarrhea while taking it, your doctor may reduce the dose, for example. B. one capsule (100 mg) once or twice a day. Your doctor will tell you how long your treatment will last.
