Pharmakon1600-01503237

• 分子结构分类: 有机化合物 - 脂质和类脂质分子 - 类固醇和类固醇衍生物
• 英文名称: Pharmakon1600-01503237
• 英文别名: (4R,8S)-8-(2-chloroacetyl)-12-fluoro-11-hydroxy-6,6,9,13-tetramethyl-5,7-dioxapentacyclo[10.8.0.02,9.04,8.013,18]icos-17-en-16-one
• 化学式: C₂₄H₃₂ClFO₅
• 分子量: 455.0
• InChiKey: MUQNGPZZQDCDFT-RLDTZFIDSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  3.6
• 重原子数：
  31
• 可旋转键数：
  2
• 环数：
  5.0
• sp3杂化的碳原子比例：
  0.83
• 拓扑面积：
  72.8
• 氢给体数：
  1
• 氢受体数：
  6

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：US20120021487A1

  公开（公告）日：2012-01-26

  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 LYSOSOMAL STORAGE DISORDERS
  申请人：The Hospital for Sick Children

  公开号：US20140235713A1

  公开（公告）日：2014-08-21

  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.