Erschienen in:
01.11.2003 | Adis Drug Profile
Miglustat
A Viewpoint by Frances M. Platt and Terry D. Butters
verfasst von:
Frances M. Platt, Terry D. Butters
Erschienen in:
Drugs
|
Ausgabe 22/2003
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Excerpt
Miglustat is a polyhydroxylated alkaloid or imino sugar that is a structural analogue of glucose. It inhibits the enzyme ceramide glucosyltransferase that catalyses the first step in glucosphingolipid biosynthesis. Glucosphingolipids are stored in several lysosomal storage diseases, including type 1 Gaucher’s disease. Miglustat has the potential to reduce synthesis of these molecules to offset the catabolic defect characteristic of these disorders. …