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Indications for Zavesca
– Adult patients with mild to moderate Gaucher disease type 1 who are not candidates for enzyme replacement therapy;
– Treatment of progressive neurological symptoms in adults and children with Niemann-Pick disease type C.
In Gaucher disease type 1, due to inherited metabolic disorders, the natural degradation of glucosylceramide does not occur, which leads to the accumulation of this substance in lysosomes and is accompanied by pathological changes in many organs. Miglustat has an inhibitory effect on glucosylceramide synthase, which is the enzyme responsible for the first step in the synthesis of most glycolipids. The inhibitory effect of glucosylceramide synthase is the basis for substrate-lowering therapy in Gaucher disease.
Niemann-Pick disease type C (sphingomyelin lipidosis) is a very rare disease that develops as a result of impaired intracellular lipid transport and manifests itself in neurodegenerative changes. The disease is characterized by an invariably progressive course and high mortality. It is believed that neurological changes in Niemann-Pick disease type C develop secondarily due to the accumulation of glycosphingolipids in neurons and glial cells.
Active ingredient: miglustat
Prescription medicine
