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Compound Summary for: CID 51634

miglustat

Also known as: Zavesca; NB-DNJ; N-Butylmoranoline; N-Butyldeoxynojirimycin; n-Butyl deoxynojirimycin; BuDNJ; Butyldeoxynojirimycin; miglustatum
Molecular Formula: C10H21NO4   Molecular Weight: 219.27804   InChIKey: UQRORFVVSGFNRO-UTINFBMNSA-N
Miglustat is a drug used to treat Gaucher disease. It inhibits the enzyme glucosylceramide synthase, an essential enzyme for the synthesis of most glycosphingolipids. It is only used for patients who cannot be treated with enzyme replacement therapy with imiglucerase. Miglustat is marketed under the trade name Zavesca. Miglustat is now the first and only approved therapy for patients with Niemann-Pick disease type C (NP-C). It has recently been approved for treatment of progressive neurological symptoms in adult and pediatric patients in the European Union, Brazil, and South Korea. Miglustat was first developed as an anti-HIV agent in the 1990s. However, clinical experience with miglustat showed that therapeutic levels of the drug could not be achieved in patients without a high incidence of adverse effect.   From: DrugBank
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