Pharmakon1600-01504273

• 分子结构分类: 有机化合物 - 有机杂环化合物 - 哌啶类
• 英文名称: Pharmakon1600-01504273
• 英文别名: (4R,5R)-1-(2-hydroxyethyl)-2-(hydroxymethyl)piperidine-3,4,5-triol
• 化学式: C₈H₁₇NO₅
• 分子量: 207.22
• InChiKey: IBAQFPQHRJAVAV-WMEPKMGJSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  -2.6
• 重原子数：
  14
• 可旋转键数：
  3
• 环数：
  1.0
• sp3杂化的碳原子比例：
  1.0
• 拓扑面积：
  104
• 氢给体数：
  5
• 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：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：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.