Miglustat
Gaucher Disease
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Drug Status
Government Approvals
US(FDA), UK(BNF)
WHO Essential Medicine
NO
Known Teratogen
No
Pharmaceutical Class
Enzyme Stabilizer and Glucosylceramide Synthase Inhibitor
Controlled Drug Substance
No
Miglustat
Summary
Miglustat is used for the treatment of Gaucher disease type 1 and Pompe disease. These are genetic conditions that cause certain fatty substances to accumulate in the body, leading to various health issues.
Miglustat works by inhibiting the production of certain fatty substances in the body. This reduces their buildup in organs and tissues, which helps manage symptoms associated with Gaucher disease type 1 and Pompe disease.
For adults with Gaucher disease type 1, Miglustat is typically taken up to three times a day. For Pompe disease, it is taken every other week one hour before cipaglucosidase alfaatga. Always follow your doctor's prescription.
Common side effects of Miglustat include diarrhea, stomach pain, gas, loss of appetite, weight loss, upset stomach, vomiting, constipation, indigestion, dry mouth, weakness, muscle cramps, dizziness, nervousness, headache, and memory problems.
Miglustat is not recommended during pregnancy as it may harm the fetus. It may also damage sperm, affecting male fertility. Patients with kidney disease or nervous system disorders should use it with caution. Always consult a doctor before use.
Indications and Purpose
How does Miglustat work?
Miglustat works by inhibiting the production of certain fatty substances in the body, reducing their buildup in organs and tissues. This helps manage symptoms associated with Gaucher disease type 1 and Pompe disease, improving patient outcomes.
Is Miglustat effective?
Miglustat is used to treat Gaucher disease type 1 and Pompe disease by preventing the body from producing certain fatty substances, reducing their buildup and associated symptoms. Clinical trials have shown its effectiveness in managing these conditions.
What is Miglustat?
Miglustat is used to treat Gaucher disease type 1 and Pompe disease. It works by inhibiting the production of certain fatty substances, reducing their buildup in the body and alleviating symptoms. It is an enzyme inhibitor that helps manage these genetic conditions.
Directions for Use
For how long do I take Miglustat?
Miglustat is used as a long-term treatment for Gaucher disease type 1 and Pompe disease. It controls symptoms but does not cure the diseases, so it is typically taken continuously as prescribed by a doctor.
How do I take Miglustat?
Miglustat can be taken with or without food for Gaucher disease, with plenty of water. For Pompe disease, take it with unsweetened beverages like water, coffee, or tea, at least 2 hours before or after eating or drinking other beverages. Follow your doctor's instructions.
How should I store Miglustat?
Store Miglustat in its original container, tightly closed, at room temperature, away from excess heat and moisture. Keep it out of reach of children. Do not flush it down the toilet; use a medicine take-back program for disposal.
What is the usual dose of Miglustat?
For adults with Gaucher disease type 1, Miglustat is typically taken up to three times a day. For Pompe disease, it is taken every other week, one hour before cipaglucosidase alfa-atga. The dosage for children is not specified in the provided content, and it is important to follow a doctor's prescription.
Warnings and Precautions
Can Miglustat be taken safely while breastfeeding?
Breastfeeding is not recommended while taking Miglustat, as it may lead to serious adverse reactions in breastfed infants. Consult a doctor for advice on feeding options during treatment.
Can Miglustat be taken safely while pregnant?
Miglustat is contraindicated during pregnancy as it may cause harm to the fetus. Women of reproductive potential should use effective contraception during treatment and for at least 60 days after the last dose. If pregnancy occurs, contact a doctor immediately.
Is Miglustat safe for the elderly?
Clinical trials did not include enough patients aged 65 and older to determine if they respond differently from younger adults. Elderly patients should use Miglustat under medical supervision.
Who should avoid taking Miglustat?
Miglustat is contraindicated during pregnancy due to potential harm to the fetus. It may also damage sperm, affecting male fertility. Patients with kidney disease or nervous system disorders should use it with caution. Consult a doctor before use.
