API reference
Miglustat
- CAS number
- 72599-27-0
- Molecular formula
- C10H21NO4
- Molecular weight
- 219.28 g/mol
About this molecule
Miglustat, commonly marketed under the trade name Zavesca, 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 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.
Source: DrugBank, via PubChem.
Identifiers
- IUPAC name
- (2R,3R,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol
- InChIKey
- UQRORFVVSGFNRO-UTINFBMNSA-N
- SMILES
- CCCCN1C[C@@H]([C@H]([C@@H]([C@H]1CO)O)O)O
- Other CAS numbers recorded
- 134282-77-2
- PubChem CID
- 51634
Also known as
Miglustat · Zavesca · N-Butyldeoxynojirimycin · NB-DNJ · Butyldeoxynojirimycin · N-butyl-1-deoxynojirimycin · N-Butylmoranoline · BuDNJ
Chemical data on this page is drawn from PubChem (National Library of Medicine) and is provided for identification only. Hanpars does not hold stock of every substance listed; availability, grade, documentation and regulatory status are confirmed per enquiry.
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