Abbreviated name: IFM
Synonyms: iso-fagomine | isofagomine
Compound class:
Synthetic organic
Ligand Activity Visualisation ChartsThese are box plot that provide a unique visualisation, summarising all the activity data for a ligand taken from ChEMBL and GtoPdb across multiple targets and species. Click on a plot to see the median, interquartile range, low and high data points. A value of zero indicates that no data are available. A separate chart is created for each target, and where possible the algorithm tries to merge ChEMBL and GtoPdb targets by matching them on name and UniProt accession, for each available species. However, please note that inconsistency in naming of targets may lead to data for the same target being reported across multiple charts. ✖ |
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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. |