(3R,4R,5R)-5-(Hydroxymethyl)-1-(4-pyridinylmethyl)-3,4-piperidinediol | 1347751-17-0

• 分子结构分类: 有机化合物 - 有机氮化合物
• 英文名称: (3R,4R,5R)-5-(Hydroxymethyl)-1-(4-pyridinylmethyl)-3,4-piperidinediol
• 英文别名: (3R,4R,5R)-5-(hydroxymethyl)-1-(pyridin-4-ylmethyl)piperidine-3,4-diol
• CAS: 1347751-17-0
• 化学式: C₁₂H₁₈N₂O₃
• 分子量: 238.28
• InChiKey: HEUKMGNKIWGRQK-IJLUTSLNSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  -0.6
• 重原子数：
  17
• 可旋转键数：
  3
• 环数：
  2.0
• sp3杂化的碳原子比例：
  0.58
• 拓扑面积：
  76.8
• 氢给体数：
  3
• 氢受体数：
  5

文献信息

• METHOD FOR THE TREATMENT OF NEUROLOGICAL DISORDERS BY ENHANCING THE ACTIVITY OF BETA-GLUCOCEREBROSIDASE
  申请人：Wustman Alan Brandon

  公开号：US20080009516A1

  公开（公告）日：2008-01-10

  Provided is a method of increasing the stability of wild-type β-glucocerebrosidase. Also provided are methods of treating and/or preventing an individual having a neurological disease in which increased expression or activity of β-glucocerebrosidase in the central nervous system would be beneficial. This method comprises administering an effective amount of a pharmacologic chaperone for β-glucocerebrosidase, with the proviso that the individual does not have a mutation in the gene encoding β-glucocerebrosidase. Further provided are β-glucocerebrosidase inhibitors which have been identified as specific pharmacologic chaperones and which have been shown to increase activity of β-glucocerebrosidase in vivo in the central nervous system.

  提供了一种增加野生型β-葡萄糖苷酶稳定性的方法。同时，提供了一种治疗和/或预防神经系统疾病的方法，其中在中枢神经系统中增加β-葡萄糖苷酶的表达或活性将有益。该方法包括给予β-葡萄糖苷酶的药理伴侣的有效剂量，但前提是该个体没有β-葡萄糖苷酶编码基因的突变。进一步提供了β-葡萄糖苷酶抑制剂，这些抑制剂已被确定为特定的药理伴侣，并已被证明在中枢神经系统内增加β-葡萄糖苷酶的活性。
• Method for the Treatment of Neurological Disorders by Enhancing the Activity of Beta-Glucocerebrosidase
  申请人：Amicus Therapeutics, Inc.

  公开号：US20150025109A1

  公开（公告）日：2015-01-22

  Provided is a method of increasing the stability of wild-type β-glucocerebrosidase. Also provided are methods of treating and/or preventing an individual having a neurological disease in which increased expression or activity of β-glucocerebrosidase in the central nervous system would be beneficial. This method comprises administering an effective amount of a pharmacologic chaperone for β-glucocerebrosidase, with the proviso that the individual does not have a mutation in the gene encoding β-glucocerebrosidase. Further provided are β-glucocerebrosidase inhibitors which have been identified as specific pharmacologic chaperones and which have been shown to increase activity of β-glucocerebrosidase in vivo in the central nervous system.

  提供了一种增加野生型β-葡萄糖苷酶稳定性的方法。还提供了治疗和/或预防神经系统疾病的方法，其中在中枢神经系统中增加β-葡萄糖苷酶的表达或活性将有益。该方法包括给予β-葡萄糖苷酶药理伴侣的有效剂量，前提是个体没有β-葡萄糖苷酶编码基因的突变。此外，还提供了已被确定为特定药理伴侣并已被证明能够在中枢神经系统中增加β-葡萄糖苷酶活性的β-葡萄糖苷酶抑制剂。
• Method for the treatment of neurological disorders by enhancing the activity of β-glucocerebrosidase
  申请人：Amicus Therapeutics, Inc.

  公开号：US10064851B2

  公开（公告）日：2018-09-04

  Provided is a method of increasing the stability of wild-type β-glucocerebrosidase. Also provided are methods of treating and/or preventing an individual having a neurological disease in which increased expression or activity of β-glucocerebrosidase in the central nervous system would be beneficial. This method comprises administering an effective amount of a pharmacologic chaperone for β-glucocerebrosidase, with the proviso that the individual does not have a mutation in the gene encoding β-glucocerebrosidase. Further provided are β-glucocerebrosidase inhibitors which have been identified as specific pharmacologic chaperones and which have been shown to increase activity of β-glucocerebrosidase in vivo in the central nervous system.

  本文提供了一种提高野生型β-葡糖脑苷脂稳定性的方法。还提供了治疗和/或预防神经系统疾病患者的方法，在这种疾病中，增加中枢神经系统中β-葡糖脑苷脂的表达或活性是有益的。该方法包括施用有效量的β-葡糖脑苷脂药理伴侣，但前提是个体没有β-葡糖脑苷脂编码基因的突变。此外，还提供了β-葡糖脑抑制剂，这些抑制剂已被确定为特异性药理合剂，并已被证明能提高β-葡糖脑在体内中枢神经系统中的活性。
• METHOD FOR THE TREATMENT OF NEUROLOGICAL DISORDERS BY ENHANCING THE ACTIVITY OF BETA -GLUCOCEREBROSIDASE
  申请人：Amicus Therapeutics, Inc.

  公开号：EP2040548B1

  公开（公告）日：2012-05-02
• US7829579B2
  申请人：——

  公开号：US7829579B2

  公开（公告）日：2010-11-09