Glucosylceramide synthase inhibitor
- Substrate-reduction therapy
- Less glycosphingolipid made → less accumulates in Gaucher disease
Drugs with this mechanism (1)
- Eliglustat — Glucosylceramide synthase inhibitor for Gaucher disease.
Self-check
- Is glucosylceramide synthase inhibition a substrate-reduction strategy?
Yes. - In which disease does reduced glycosphingolipid synthesis reduce accumulation?
Gaucher disease.
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