2-[4-[4-(2-Methoxyethoxy)phenyl]phenyl]propan-2-ylcarbamic acid

• 分子结构分类: 有机化合物 - 苯类化合物 - 苯和取代衍生物
• 英文名称: 2-[4-[4-(2-Methoxyethoxy)phenyl]phenyl]propan-2-ylcarbamic acid
• 英文别名: 2-[4-[4-(2-methoxyethoxy)phenyl]phenyl]propan-2-ylcarbamic acid
• 化学式: C₁₉H₂₃NO₄
• 分子量: 329.4
• InChiKey: OYNKMHNCZBJLRP-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  3.2
• 重原子数：
  24
• 可旋转键数：
  7
• 环数：
  2.0
• sp3杂化的碳原子比例：
  0.32
• 拓扑面积：
  67.8
• 氢给体数：
  2
• 氢受体数：
  4

文献信息

• [EN] METHODS FOR TREATING NEUROLOGICAL SYMPTOMS ASSOCIATED WITH LYSOSOMAL STORAGE DISEASES<br/>[FR] MÉTHODES DE TRAITEMENT DE SYMPTÔMES NEUROLOGIQUES ASSOCIÉS À DES MALADIES LYSOSOMALES
  申请人：GENZYME CORP

  公开号：WO2021156769A1

  公开（公告）日：2021-08-12

  Methods are provided for treating or preventing neurological symptoms and disorders which are associated with, e.g., lysosomal storage diseases. The methods include enhancing neuronal connectivity within the brain of a subject, increasing brain tissue volume, or preventing or delaying loss of brain tissue volume in a subject. Also provided are methods for monitoring the progression or regression of a neurological disorder, or assessing the onset of a neurological disorder, associated with a lysosomal storage disease, in which brain tissue volume of the subject is measured.

  提供了用于治疗或预防与溶酶体贮积病等相关的神经症状和疾病的方法。这些方法包括增强受试者大脑内的神经连接、增加大脑组织体积，或者预防或延迟受试者大脑组织体积的丧失。还提供了用于监测神经疾病的进展或退化，或评估与溶酶体贮积病相关的神经疾病的发作的方法，其中测量了受试者的大脑组织体积。
• [EN] METHODS FOR TREATING PROTEINOPATHIES<br/>[FR] PROCÉDÉS DE TRAITEMENT DE PROTÉINOPATHIES
  申请人：GENZYME CORP

  公开号：WO2016145046A1

  公开（公告）日：2016-09-15

  This disclosure relates to a method of treating a proteinopathy in a subject, the method comprising administering to the subject an effective amount of a quinuclidine compound. The disclosure also relates to a method of reducing, reversing or preventing the accumulation of protein aggregates in tissue of a subject diagnosed as having a proteinopathy, or being at risk of developing a proteinopathy, the method comprising administering to the subject an effective amount of a quinuclidine compound. Also disclosed is a pharmaceutical composition comprising a quinuclidine compound for use in said methods. The proteinopathy may be a synucleinopathy or a tauopathy, such as Parkinson's disease, Alzheimer's disease or dementia with Lewy bodies.

  这份披露涉及一种治疗受试者蛋白病的方法，该方法包括向受试者施用有效量的环丙胺化合物。该披露还涉及一种减少、逆转或预防被诊断为患有蛋白病或有患蛋白病风险的受试者组织中蛋白聚集物积累的方法，该方法包括向受试者施用有效量的环丙胺化合物。还披露了一种包含环丙胺化合物的药物组合物，用于上述方法。蛋白病可能是突触核蛋白病或tau蛋白病，如帕金森病、阿尔茨海默病或带有Lewy小体的痴呆症。
• [EN] GLUCOSYLCERAMIDE SYNTHASE INHIBITORS<br/>[FR] INHIBITEURS DE SYNTHASE DE GLUCOSYLCÉRAMIDE
  申请人：GENZYME CORP

  公开号：WO2014043068A1

  公开（公告）日：2014-03-20

  The invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment of metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, cystic disease and for the treatment of cancer.

  该发明涉及抑制葡萄糖鞘氨醇合成酶（GCS）的抑制剂，用于治疗代谢性疾病，如溶酶体贮积病，单独或与酶替代疗法、囊性疾病以及癌症的联合治疗。
• [EN] METHODS FOR REDUCING GLYCOSPHINGOLIPID CONCENTRATION IN BRAIN TISSUE AND METHODS OF TREATMENT OF NEURODEGENERATIVE DISEASES INVOLVING THE SAME<br/>[FR] PROCÉDÉS DE RÉDUCTION DE LA CONCENTRATION EN GLYCOSPHINGOLIPIDES DANS LE TISSU CÉRÉBRAL ET PROCÉDÉS DE TRAITEMENT DE MALADIES NEURODÉGÉNÉRATIVES IMPLIQUANT CES DERNIERS
  申请人：GENZYME CORP

  公开号：WO2022024062A1

  公开（公告）日：2022-02-03

  Provided are methods for treating or preventing Parkinson's disease or dementia with Lewy Bodies in a human subject. The methods target lipid dysregulation in brain tissue of the subject and can, in particular, reduce the concentration of glycosphingolipids in brain tissue. The methods use quinuclidine compounds of formula (I), or pharmaceutically acceptable prodrugs or salts thereof. In particular, the human subject may be a carrier of one or more glucocerebrosidase 1 gene (GBA1) mutations, e.g. a heterozygous carrier of GBA1 mutations. Also provided are methods for assessing the effectiveness of treatments as described herein.

  提供了一种治疗或预防人类患帕金森病或Lewy体痴呆的方法。该方法针对患者脑组织中的脂质失调，并可特别降低脑组织中的糖脂类浓度。该方法使用公式（I）的喹啉类化合物或其药学上可接受的前药或盐。特别地，人类受试者可能是一种或多种葡萄糖酸酰基酶1基因（GBA1）突变的携带者，例如GBA1突变的杂合携带者。此外，还提供了评估本文所述治疗方法有效性的方法。
• Biomarkers of polycystic kidney disease and uses thereof
  申请人：Genzyme Corporation

  公开号：US10871495B2

  公开（公告）日：2020-12-22

  Provided herein are methods for determining the efficacy of treatment for polycystic kidney disease (PKD) in a patient, diagnosing PKD in a patient, staging PKD in a patient, and monitoring PKD in a patient. These methods include determining a single or multiple levels of one or more markers selected from the group of Proliferating Cell Nuclear Antigen (PCNA), cyclin D1, cyclin D3, MAPKERK kinase 1 (MEK), ribosomal protein S6 (S6), phosphorylated ribosomal protein S6 (pS6), extracellular signal-regulated kinase (ERK), phosphorylated extracellular signal-regulated kinase (pERK), protein kinase B (Akt), phosphorylated protein kinase B (pAkt), caspase-2, total S6, and retinoblastoma binding protein (RBBP). Also provided are kits that include at least three antibodies that specifically bind to one or more of these markers.

  本文提供了用于确定患者多囊肾病（PKD）疗效、诊断患者多囊肾病、对患者多囊肾病进行分期以及监测患者多囊肾病的方法。这些方法包括测定选自增殖细胞核抗原 (PCNA)、细胞周期蛋白 D1、细胞周期蛋白 D3、MAPKERK 激酶 1 (MEK)、核糖体蛋白 S6 (S6)、磷酸化核糖体蛋白 S6 (pS6) 组的一种或多种标记物的单个或多个水平、细胞外信号调节激酶 (ERK)、磷酸化细胞外信号调节激酶 (pERK)、蛋白激酶 B (Akt)、磷酸化蛋白激酶 B (pAkt)、Caspase-2、总 S6 和视网膜母细胞瘤结合蛋白 (RBBP)。此外，还提供了包括至少三种抗体的试剂盒，这些抗体能特异性地与这些标记物中的一种或多种结合。