(1R,4S,5'S,6R,6'S,8R,10E,12S,13S,14E,16E,20R,24S)-6'-cyclohexyl-24-hydroxy-12-[(4S,6S)-5-hydroxy-4-methoxy-6-methyloxan-2-yl]oxy-5',11,13,22-tetramethylspiro[3,7,19-trioxatetracyclo[15.6.1.14,8.020,24]pentacosa-10,14,16,22-tetraene-6,2'-oxane]-2-one

• 分子结构分类: 有机化合物 - 苯丙烷和聚酮 - 大环内酯类和类似物
• 英文名称: (1R,4S,5'S,6R,6'S,8R,10E,12S,13S,14E,16E,20R,24S)-6'-cyclohexyl-24-hydroxy-12-[(4S,6S)-5-hydroxy-4-methoxy-6-methyloxan-2-yl]oxy-5',11,13,22-tetramethylspiro[3,7,19-trioxatetracyclo[15.6.1.14,8.020,24]pentacosa-10,14,16,22-tetraene-6,2'-oxane]-2-one
• 化学式: C₄₃H₆₄O₁₀
• 分子量: 741.0
• InChiKey: LNASWLBIATYITH-ZEYMBTCOSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  5.5
• 重原子数：
  53
• 可旋转键数：
  4
• 环数：
  7.0
• sp3杂化的碳原子比例：
  0.79
• 拓扑面积：
  122
• 氢给体数：
  2
• 氢受体数：
  10

文献信息

• Method for enhancing folding and transport of misfolded glucocerebrosidase
  申请人：The Hospital for Sick Children

  公开号：US10028922B2

  公开（公告）日：2018-07-24

  Therapeutic compositions and methods for treatment of late-onset Gaucher disease are described herein. The compositions comprise compounds having activity as pharmacological chaperones for mutant forms of the beta-glucocerebrosidase. Methods of treatment involve providing therapeutically effective amounts of such compositions to subjects in need thereof.

  本文描述了治疗晚发型戈谢病的治疗组合物和方法。这些组合物包括对突变形式的β-葡糖脑苷脂具有药理伴侣活性的化合物。治疗方法包括向有需要的受试者提供治疗有效量的此类组合物。
• Method of treating gaucher disease
  申请人：Mahuran Don J.

  公开号：US20090075960A1

  公开（公告）日：2009-03-19

  Therapeutic compositions and methods for treatment of late-onset Gaucher disease are described herein. The compositions comprise compounds having activity as pharmacological chaperones for mutant forms of the beta-glucocerebrosidase. Methods of treatment involve providing therapeutically effective amounts of such compositions to subjects in need thereof.
• METHOD OF TREATING GAUCHER DISEASE
  申请人：The Hospital For Sick Children

  公开号：US20130090388A1

  公开（公告）日：2013-04-11

  Therapeutic compositions and methods for treatment of late-onset Gaucher disease are described herein. The compositions comprise compounds having activity as pharmacological chaperones for mutant forms of the beta-glucocerebrosidase. Methods of treatment involve providing therapeutically effective amounts of such compositions to subjects in need thereof.
• METHOD FOR ENHANCING FOLDING AND TRANSPORT OF MISFOLDED GLUCOCEREBROSIDASE
  申请人：The Hospital for Sick Children

  公开号：US20140249233A1

  公开（公告）日：2014-09-04

  Therapeutic compositions and methods for treatment of late-onset Gaucher disease are described herein. The compositions comprise compounds having activity as pharmacological chaperones for mutant forms of the beta-glucocerebrosidase. Methods of treatment involve providing therapeutically effective amounts of such compositions to subjects in need thereof.
• Method For Enhancing Folding and Transport of Misfolded Glucocerebrosidase
  申请人：The Hospital for Sick Children

  公开号：US20160113892A1

  公开（公告）日：2016-04-28

  Therapeutic compositions and methods for treatment of late-onset Gaucher disease are described herein. The compositions comprise compounds having activity as pharmacological chaperones for mutant forms of the beta-glucocerebrosidase. Methods of treatment involve providing therapeutically effective amounts of such compositions to subjects in need thereof.