| Name | Eliglustat hemitartrate |
| Description | Eliglustat hemitartrate (Genz-112638) , with an IC50 of 24 nM, is a specific, potent, oral active glucocerebral glycosidase inhibitor. |
| In vitro | Eliglustat tartrate demonstrates strong potency, with an IC50 of 24 nM, and high specificity against the target enzyme. |
| In vivo | A novel inhibitor of glucosylceramide synthase (Genz-112638) and assessed its activity in a murine model of Gaucher disease (D409V/null).?Biochemical characterization of Genz-112638 showed good potency (IC(50) approximately 24nM) and specificity against the target enzyme.?Mice that received drug prior to significant accumulation of substrate (10 weeks of age) showed reduced levels of glucosylceramide and number of Gaucher cells in the spleen, lung and liver when compared to age-matched control animals.?Treatment of older mice that already displayed significant amounts of tissue glucosylceramide (7 months old) resulted in arrest of further accumulation of the substrate and appearance of additional Gaucher cells in affected organs.?These data indicate that substrate inhibition therapy with Genz-112638 represents a viable alternate approach to enzyme therapy to treat the visceral pathology in Gaucher disease. |
| Storage | Shipping with blue ice/Shipping at ambient temperature. |
| Solubility Information | H2O : 50 mg/mL (104.26 mM), Sonication is recommended.
10% DMSO+40% PEG300+5% Tween 80+45% Saline : 2 mg/mL (4.17 mM), Sonication is recommended.
DMSO : 50 mg/mL (104.26 mM), Sonication is recommended.
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| Keywords | Inhibitor | inhibit | glucocerebroside synthase | Genz112638 | Genz 112638 | Eliglustat Tartrate | Eliglustat hemitartrate | Eliglustat |
| Inhibitors Related | Alendronate sodium trihydrate | Serotonin hydrochloride | Serotonin | Rosmarinic acid | D(+)-Raffinose pentahydrate | Risedronic Acid | 3-Methoxybenzamide | Entacapone | CBHcy | (-)-Gallocatechin gallate | Flopropione | Tolcapone |
| Related Compound Libraries | FDA-Approved & Pharmacopeia Drug Library | Bioactive Compound Library | Approved Drug Library | EMA Approved Drug Library | Drug Repurposing Compound Library | Inhibitor Library | NO PAINS Compound Library | FDA-Approved Drug Library | Orally Active Compound Library | Clinical Compound Library | Bioactive Compounds Library Max |
United States
