| Description | Eliglustat (Genz 99067) is an oral inhibitor of glucosylceramide synthase which is used in the therapy of type 1 Gaucher disease. |
| In vitro | Eliglustat Tartrate啮齿类动物体内快速代谢,其体内半衰期在15-45分钟.对正常小鼠、大鼠、狗中通过静脉注射和口服途径给药（Eliglustat Tartrate）,脾脏、肾脏和肝脏中葡糖神经酰胺的含量显著下降. |
| In vivo | Eliglustat tartrate是葡萄糖神经酰胺合成酶的抑制剂,对该酶的抑制作用高度特异,对其他多种糖苷酶的抑制作用有限甚至没有抑制活性。 |
| Cell experiments | The inhibitory activity of Genz-112638 is determined indirectly by measuring its effect on the cell surface levels of the gangliosides GM1 and GM3 on either K562 or B16/F10 cells. GM1 levels on the K562 cells are determined by incubating the cells with increasing amounts of Genz-112638 (0.6-1000 nM) for 72 h after which the cells are harvested and stained using 10 μg of recombinant cholera toxin-FITC in 100 μL phosphate buffered saline (PBS) containing 0.5% bovine serum albumin (BSA) for 30 min on ice. Cells are ished, resuspended in PBS containing 0.5% BSA and the fluorescence quantitated[1]. |
| Target activity | glucosylceramide synthase (GCS):20 nM |
| Synonyms | GENZ-112638, Genz 99067, 依利格鲁司特 |
| molecular weight | 404.54 |
| Molecular formula | C23H36N2O4 |
| CAS | 491833-29-5 |
| Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year |
| Solubility | Ethanol: 74 mg/mL (182.9 mM) DMSO: 45 mg/mL (111.24 mM) H2O: < 1 mg/mL (insoluble or slightly soluble) |
| References | 1. Shayman JA. Drugs Future. 2010, 35(8):613-620. 2. Nelson S. Yew, et al. PLoS One. 2010, 5(6):e11239. |