Miglustat hydrochloride

• CAT Number: I002616
• CAS Number: 210110-90-0
• Molecular Formula: C10H21NO4 • HCl
• Molecular Weight: 255.74
• Purity: ≥95%
• Synonyms: Miglustat;NB-DNJ
• Target: Glucosylceramide Synthase (GCS)
• Solubility: H2O: ≥ 34 mg/mL
• Storage: -20°C
• IUPAC Name: (2R,3R,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol;hydrochloride
• InChI: InChI=1S/C10H21NO4.ClH/c1-2-3-4-11-5-8(13)10(15)9(14)7(11)6-12;/h7-10,12-15H,2-6H2,1H3;1H/t7-,8+,9-,10-;/m1./s1
• InChIKey: QPAFAUYWVZMWPR-ZSOUGHPYSA-N
• SMILES: CCCCN1C[C@@H]([C@H]([C@@H]([C@H]1CO)O)O)O.Cl

Miglustat Hydrochloride(CAT: I002616) is a potent inhibitor of glucosylceramide synthase, the enzyme responsible for the first step in glycosphingolipid synthesis. By reducing the production of glucosylceramide, it addresses the accumulation of glycosphingolipids characteristic of Type I Gaucher disease (GD1), a lysosomal storage disorder. This oral therapy offers a substrate reduction approach, complementing enzyme replacement therapies. Miglustat Hydrochloride is also utilized in research to explore glycosphingolipid metabolism and its role in other diseases, such as Niemann-Pick disease type C. Its mechanism makes it an essential tool for advancing the understanding and treatment of metabolic and lysosomal storage disorders.