miglustat
Information, Symptoms, Treatments and Resources
What is miglustat?
Generic Name: miglustat
Other Brand Names:
Zavesca
Miglustat may reduce the formation of the protein glucosylceramide in the body. Individuals with
type 1 Gaucher disease (a hereditary disorder) are missing an enzyme to break down glucosylceramide
in the body. Therefore, it may build up in the tissues and cause problems. Untreated Gaucher disease
may result in anemia (low level of red blood cells), thrombocytopenia (low level of platelets), bone
disease, hepatomegaly (enlargement of the liver), or splenomegaly (enlargement of the spleen).
Miglustat is used in the treatment of mild to moderate type 1 Gaucher disease by people who
cannot receive enzyme replacement therapy.
Miglustat may also be used for purposes other than those listed here.
