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Abbreviated name: IFM
Synonyms: iso-fagomine | isofagomine
Compound class:
Synthetic organic
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No information available. |
Summary of Clinical Use ![]() |
Afegostat is an experimental drug for the treatment Gaucher's disease, which is caused by mutations in the β-glucocerebrosidase gene (GBA, P04062). This drug (as isofagomine tartrate) has been granted orphan status by the European Medicines Agency for this indication. |
Mechanism Of Action and Pharmacodynamic Effects ![]() |
Afegostat binds β-glucocerebrosidase (GBA, P04062), a lysosomal enzyme that hydrolyses glucocerebroside as a step in glycolipid metabolism. |