Miglustat
Miglustat
Miglustat (pronounced as mi-gloo-stat) is a medication used in the treatment of Gaucher's disease and Niemann-Pick disease. It is a small molecule inhibitor of the enzyme glucosylceramide synthase, which is involved in the production of a type of fat that accumulates in the cells of people with these diseases.
Etymology
The name "Miglustat" is derived from its chemical name, N-butyldeoxynojirimycin. The "Miglu-" part of the name comes from "miglitol," a similar compound, and the "-stat" suffix is commonly used in drug names to indicate that the drug inhibits a particular process or substance.
Usage
Miglustat is used as a first-line treatment for adults with mild to moderate type 1 Gaucher's disease who are not suitable for enzyme replacement therapy. It is also used for the treatment of progressive neurological symptoms in adults and pediatric patients with Niemann-Pick disease type C.
Mechanism of Action
Miglustat works by inhibiting the enzyme glucosylceramide synthase, which is involved in the production of glucosylceramide. This reduces the amount of glucosylceramide that is produced, which in turn reduces the amount of this substance that can accumulate in cells.
Side Effects
Common side effects of Miglustat include diarrhea, weight loss, and tremor. Less common side effects can include anemia, thrombocytopenia, and neuropathy.
Related Terms
External links
- Medical encyclopedia article on Miglustat
- Wikipedia's article - Miglustat
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