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miglustat (Acetlion, Zavesca, Opfolda)
Tradename: Acetlion (FDA approved 2003)
Indications:
- Gaucher disease
- patients who cannot receive enzyme therapy (FDA)
- Pompe disease in combination with cipaglucosidase alfa-atga (Pombiliti)
Adverse effects: GI distress, peripheral neuropathy, weight loss
Mechanism of action:
-> glucosylceramide synthase inhibitor
General
enzyme inhibitor
metabolic agent (metabolic modifier)
Properties
INHIBITS: ceramide glucosyltransferase
Database Correlations
PUBCHEM cid=51634
References
- Prescriber's Letter 11(2):suppl 2004
- Zimmerman, Ari