Overview
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Generic Name: Miglustat
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Trade Name: Zavesca
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Drug Class: Substrate reduction therapy (SRT); glucosylceramide synthase inhibitor
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Administration: Oral capsules
Indications
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Gaucher disease type 1 (mild to moderate severity) for patients unable or unwilling to receive enzyme replacement therapy (ERT).
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Niemann–Pick Disease Type C (NPC), to slow neurological progression.
Mechanism of Action
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Miglustat is an iminosugar (N-alkylated imino sugar) derivative.
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Acts by competitively inhibiting glucosylceramide synthase enzyme.
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Reduces glycosphingolipid accumulation by decreasing substrate production.
