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Eliglustat

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Chemical Structure| 491833-29-5 同义名 : Genz 99067;GENZ-112638
CAS号 : 491833-29-5
货号 : A848004
分子式 : C23H36N2O4
纯度 : 99%+
分子量 : 404.543
MDL号 : MFCD19443735
存储条件：

粉末 Sealed in dry,2-8°C

液体 -20°C:3-6个月-80°C:12个月
溶解度 :

DMSO: 105 mg/mL(259.55 mM)，配合低频超声助溶，注意：DMSO长时间开封后，会吸水并导致溶解能力下降，请避免使用长期开封的DMSO
动物实验配方：
生物活性
描述 Eliglustat is a selective inhibitor of glucosylceramide synthase that is extensively metabolized by CYP2D6 and, to a lesser extent by CYP3A4; it is also an inhibitor of the P-gp transporter[3]. Eliglustat is an oral inhibitor of glucosylceramide synthase which is used in the therapy of type 1 Gaucher disease[4]. Eliglustat inhibits glucosylceramide synthase, thereby decreasing production of the substrate glucosylceramide and reducing its accumulation[5]. Administration of eliglustat to Swiss CD-1 mice at 0, 10, 25 or 75 mg/kg/day for 104 weeks by dietary admixture did not influence survival or bodyweight evolution, or produce any clinical indication of poor condition. Eliglustat was not carcinogenic to mice or rats in standard lifetime bioassays[6].
临床研究
NCT号 适应症或疾病 临床期 招募状态 预计完成时间 地点
NCT01074944 Gaucher Disease Phase 3 Completed - -
NCT00358150 Gaucher Disease, Type 1 ... 展开 >> Cerebroside Lipidosis Syndrome Glucocerebrosidase Deficiency Disease Glucosylceramide Beta-Glucosidase Deficiency Disease Gaucher Disease, Non-Neuronopathic Form 收起 << Phase 2 Completed - United States, New York ... 展开 >> New York University New York, New York, United States New York, New York, United States Argentina Aprillus Asistencia e Investigación Buenos Aires, Argentina Hospital de Oncologia Maria Curie Buenos Aires, Argentina IMAI Buenos Aires, Argentina Instituto Argentino de Diagnostico y Tratamiento (IADT) Buenos Aires, Argentina Buenos Aires, Argentina Hospital Ramos Mejia Ciudad Autonoma de Buenos Aires, Argentina Israel Rambam Medical Center Haifa, Israel Haifa, Israel Sha'are Zedek Medical Centre Jerusalem, Israel Jerusalem, Israel Italy Universita degli Studi di Milano Milano, Italy Mexico Instituto Mexicano del Seguro Social D.f., Mexico Mexico City, Mexico Russian Federation Hematology Research Center of Ministry of Healthcare of the Russian Federation Moscow, Russian Federation Moscow, Russian Federation 收起 <<
NCT00943111 - Completed - -
实验方案
1mg 5mg 10mg

1 mM

5 mM

10 mM

2.47mL

0.49mL

0.25mL

12.36mL

2.47mL

1.24mL

24.72mL

4.94mL

2.47mL
参考文献

[1]Shayman JA. ELIGLUSTAT TARTRATE: Glucosylceramide Synthase Inhibitor Treatment of Type 1 Gaucher Disease. Drugs Future. 2010 Aug 1;35(8):613-620.

[2]Yew NS, Zhao H, et al. Increased hepatic insulin action in diet-induced obese mice following inhibition of glucosylceramide synthase. PLoS One. 2010 Jun 21;5(6):e11239.

[3]Almeida-Calpe A, López de Frutos L, Medrano-Engay B, García-García CB, Ribate MP, Giraldo P. Metabolizing profile of the cytochrome pathway CYP2D6, CYP3A4 and the ABCB 1 transporter in Spanish patients affected by Gaucher disease. Chem Biol Interact. 2021 Aug 25;345:109527

[4]LiverTox: Clinical and Research Information on Drug-Induced Liver Injury [Internet]. Bethesda (MD): National Institute of Diabetes and Digestive and Kidney Diseases; 2012–. Eliglustat. 2018 Mar 5

[5]Balwani M, Burrow TA, Charrow J, Goker-Alpan O, Kaplan P, Kishnani PS, Mistry P, Ruskin J, Weinreb N. Recommendations for the use of eliglustat in the treatment of adults with Gaucher disease type 1 in the United States. Mol Genet Metab. 2016 Feb;117(2):95-103

[6]Dagher R, Watzinger M, Chevalier G, Thirion-Delalande C, Gervais F, Forster R. Carcinogenicity testing of eliglustat in mice and rats. Regul Toxicol Pharmacol. 2015 Oct;73(1):401-12