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CAS 928659-70-5

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Eliglustat tartrate

Description:
Eliglustat tartrate is a synthetic compound primarily used as a pharmacological agent in the treatment of Gaucher disease, a genetic disorder resulting from the deficiency of the enzyme glucocerebrosidase. It functions as a substrate reduction therapy, inhibiting the synthesis of glucosylceramide, thereby reducing its accumulation in cells. The substance is characterized by its molecular formula, which reflects its complex structure, and it typically appears as a white to off-white powder. Eliglustat tartrate is soluble in water and exhibits stability under standard storage conditions. Its mechanism of action involves selective inhibition of glucosylceramide synthase, which is crucial for its therapeutic efficacy. The compound is administered orally and has a favorable pharmacokinetic profile, allowing for once-daily dosing. As with any medication, potential side effects may include gastrointestinal disturbances and other systemic reactions, necessitating monitoring during treatment. Overall, Eliglustat tartrate represents a significant advancement in the management of Gaucher disease, providing an alternative to enzyme replacement therapy.
Formula:C23H36N2O4C4H6O6
InChI:InChI=1S/C23H36N2O4.C4H6O6/c1-2-3-4-5-6-9-22(26)24-19(17-25-12-7-8-13-25)23(27)18-10-11-20-21(16-18)29-15-14-28-20;5-1(3(7)8)2(6)4(9)10/h10-11,16,19,23,27H,2-9,12-15,17H2,1H3,(H,24,26);1-2,5-6H,(H,7,8)(H,9,10)/t19-,23-;1-,2-/m11/s1
InChI key:InChIKey=VNGQLUTVKDPUJS-VUBSCCJGSA-N
SMILES:[C@@H]([C@@H](CN1CCCC1)NC(CCCCCCC)=O)(O)C=2C=C3C(=CC2)OCCO3.[C@@H]([C@H](C(O)=O)O)(C(O)=O)O
Synonyms:
  • Eliglustat hemitartrate
  • Octanamide, N-[(1R,2R)-2-(2,3-dihydro-1,4-benzodioxin-6-yl)-2-hydroxy-1-(1-pyrrolidinylmethyl)ethyl]-, (2R,3R)-2,3-dihydroxybutanedioate (2:1)
  • Eliglustat tartrate
  • Genz 112638
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