(E)-but-2-enedioic acid;propane

• 分子结构分类: 有机化合物 - 有机酸及其衍生物 - 羧酸及其衍生物
• 英文名称: (E)-but-2-enedioic acid;propane
• 化学式: C10H20O4
• 分子量: 204.26
• InChiKey: RORLKPMXVRPUJY-SEPHDYHBSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  2.54
• 重原子数：
  14
• 可旋转键数：
  2
• 环数：
  0.0
• sp3杂化的碳原子比例：
  0.6
• 拓扑面积：
  74.6
• 氢给体数：
  2
• 氢受体数：
  4

文献信息

• Methods of treating sickle cell disease and related disorders using fumaric acid esters
  申请人：Augusta University Research Institute, Inc.

  公开号：US10813905B2

  公开（公告）日：2020-10-27

  Methods of using one or more fumaric acid esters or pharmacologically active salts, derivatives, analogues, or prodrugs thereof to increase expression of fetal hemoglobin (HbF) are disclosed. The methods typically include administering to a subject an effective amount of one or more fumaric acid esters optionally in combination or alternation with hydroxyurea to induce HbF expression in the subject in an effective amount to reduce one or more symptoms of a sickle cell disorder, a hemoglobinopathy, or a beta-thalassemia, or to compensate for a genetic mutation is the human beta-globin gene (HBB) or an expression control sequence thereof. Pharmaceutical dosage units and dosage regimes for use in the disclosed methods are also provided.

  本发明公开了使用一种或多种富马酸酯或其药理活性盐、衍生物、类似物或原药增加胎儿血红蛋白（HbF）表达的方法。这些方法通常包括向受试者施用有效量的一种或多种富马酸酯，可选择与羟基脲联合或交替使用，以诱导受试者体内有效量的 HbF 表达，从而减轻镰状细胞疾病、血红蛋白病或β-地中海贫血的一种或多种症状，或补偿人类β-球蛋白基因（HBB）或其表达控制序列的基因突变。还提供了用于所公开方法的药物剂量单位和剂量方案。
• Methods of Treating Sickle Cell Disease and Related Disorders Using Fumaric Acid Esters
  申请人：Georgia Regents Research Institute, Inc.

  公开号：US20140171504A1

  公开（公告）日：2014-06-19

  Methods of using one or more fumaric acid esters or pharmacologically active salts, derivatives, analogues, or prodrugs thereof to increase expression of fetal hemoglobin (HbF) are disclosed. The methods typically include administering to a subject an effective amount of one or more fumaric acid esters optionally in combination or alternation with hydroxyurea to induce HbF expression in the subject in an effective amount to reduce one or more symptoms of a sickle cell disorder, a hemoglobinopathy, or a beta-thalassemia, or to compensate for a genetic mutation is the human beta-globin gene (HBB) or an expression control sequence thereof. Pharmaceutical dosage units and dosage regimes for use in the disclosed methods are also provided.