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CAS 72599-27-0

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Miglustat

Description:
Miglustat is a chemical compound primarily used as a therapeutic agent for certain genetic disorders, particularly Gaucher disease and Niemann-Pick disease type C. It is classified as a glucosylceramidase inhibitor, which means it interferes with the metabolism of glycosphingolipids, thereby reducing their accumulation in cells. The molecular formula of miglustat is C₈H₁₅NO₃, and it has a molecular weight of approximately 169.22 g/mol. The compound is typically administered orally and is known for its ability to penetrate the blood-brain barrier, making it beneficial for neurological manifestations of these diseases. Miglustat is characterized by its white to off-white crystalline powder form, and it is soluble in water and organic solvents. Its mechanism of action involves the inhibition of the enzyme glucosylceramidase, which is crucial for the breakdown of glucocerebrosides. As with any medication, miglustat may have side effects, and its use should be monitored by healthcare professionals.
Formula:C10H21NO4
InChI:InChI=1S/C10H21NO4/c1-2-3-4-11-5-8(13)10(15)9(14)7(11)6-12/h7-10,12-15H,2-6H2,1H3/t7-,8+,9-,10-/m1/s1
InChI key:InChIKey=UQRORFVVSGFNRO-UTINFBMNSA-N
SMILES:C(CCC)N1[C@H](CO)[C@@H](O)[C@H](O)[C@@H](O)C1
Synonyms:
  • N-Butyl-1-deoxynojirimycin
  • 3,4,5-Piperidinetriol, 1-butyl-2-(hydroxymethyl)-, [2R-(2α,3β,4α,5β)]-
  • 3,4,5-Piperidinetriol, 1-butyl-2-(hydroxymethyl)-, (2R,3R,4R,5S)-
  • N-Butyldeoxynojirimycin
  • (2R,3R,4R,5S)-1-Butyl-2-(hydroxymethyl)-3,4,5-piperidinetriol
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