Method for treatment of glycolipid storage diseases
    6.
    发明授权
    Method for treatment of glycolipid storage diseases 失效
    糖脂储存疾病的治疗方法

    公开(公告)号:US06610703B1

    公开(公告)日:2003-08-26

    申请号:US09453754

    申请日:1999-12-02

    IPC分类号: A61P2500

    CPC分类号: A61K31/445

    摘要: A novel method is disclosed for the treatment of a patient affected with Gaucher's disease or other such glycolipid storage diseases. The method comprises administering to said patient a therapeutically effective amount of a long-chain N-alkyl derivative of deoxynojirimycin to alleviate or inhibit the glycolipid storage disease. The long-chain alkyl group has from nine to about 20 carbon atoms and preferably is nonyl or decyl.

    摘要翻译: 公开了用于治疗患有戈谢氏病或其他此类糖脂储存疾病的患者的新方法。 该方法包括向所述患者施用治疗有效量的脱氧野尻霉素的长链N-烷基衍生物以缓解或抑制糖脂储存疾病。 长链烷基具有9至约20个碳原子,优选为壬基或癸基。

    DEOXYNOJIRIMYCIN AND D-ARABINITOL ANALOGS AND METHODS OF USING
    7.
    发明申请
    DEOXYNOJIRIMYCIN AND D-ARABINITOL ANALOGS AND METHODS OF USING 有权
    去氧吉西他滨和D-阿拉伯糖醇模拟物及其使用方法

    公开(公告)号:US20070275998A1

    公开(公告)日:2007-11-29

    申请号:US11752015

    申请日:2007-05-22

    CPC分类号: C07D211/48 C07D211/46

    摘要: A compound of Formula I are provided: wherein R is: R1 is a substituted or unsubstituted alkyl group; W1-4 are independently selected from hydrogen, substituted or unsubstituted alkyl groups, substituted or unsubstituted haloalkyl groups, substituted or unsubstituted alkanoyl groups, substituted or unsubstituted aroyl groups, or substituted or unsubstituted haloalkanoyl groups; X1-5 are independently selected from H, NO2, N3, and NH2;Y is absent or is a substituted or unsubstituted C1-alkyl group, other than carbonyl; Z is selected from a bond or NH, provided that when Z is a bond, Y is absent, and provided that when Z is NH, Y is a substituted or unsubstituted C1-alkyl group, other than carbonyl. Also provided are D-arabinitol compounds, methods for preparing such compounds and compositions of such compounds, and methods of using such compounds.

    摘要翻译: 提供式I化合物:其中R是:R 1是取代或未取代的烷基; 取代或未取代的烷基,取代或未取代的烷基,取代或未取代的烷酰基,取代或未取代的芳酰基,或取代或未取代的卤代烷酰基; X 1-5独立地选自H,NO 2,N 3和NH 2; Y是 不存在或是不是羰基的取代或未取代的C 1 - 烷基; Z选自键或NH,条件是当Z为键时,Y不存在,条件是当Z为NH时,Y为取代或未取代的C 1 - 烷基,除 羰。 还提供了D-阿拉伯糖醇化合物,制备这些化合物的方法和这些化合物的组合物,以及使用这些化合物的方法。

    Method for treatment of CNS-involved lysosomal storage diseases
    9.
    发明授权
    Method for treatment of CNS-involved lysosomal storage diseases 失效
    CNS相关溶酶体贮积病治疗方法

    公开(公告)号:US5798366A

    公开(公告)日:1998-08-25

    申请号:US782321

    申请日:1997-01-13

    摘要: A method is disclosed for the in vivo treatment of patients having a lysosomal storage disease with a significant central nervous system (CNS) involvement. Said method comprises administration to said patient a small but storage-inhibitory effective amount of an N-alkyl derivative of a 1,5-iminosugar in which said alkyl group contains from about 2 to about 8 carbon atoms and said 1,5-iminosugar is 1,5-dideoxy-1,5-imino-D-glucitol, or 1,5-dideoxy-1,5-imino-D-galactitol, or an O-acylated pro-drug of said 1,5-iminosugar. In an illustrative example, CNS storage of GM2 ganglioside is inhibited in Tay-Sachs mice by administration of 1,5-(butylimino)-1,5-dideoxy-D-glucitol.

    摘要翻译: 公开了一种用于体内治疗具有显着的中枢神经系统(CNS)参与的溶酶体贮积病的患者的方法。 所述方法包括向所述患者施用小的但储存抑制的有效量的1,5-亚氨基二糖的N-烷基衍生物,其中所述烷基含有约2至约8个碳原子,所述1,5-亚氨基二糖是 1,5-二脱氧-1,5-亚氨基-D-葡萄糖醇或1,5-二脱氧-1,5-亚氨基-D-半乳糖醇,或所述1,5-亚氨基二糖的O-酰化前体药。 在说明性实例中,通过施用1,5-(丁基亚氨基)-1,5-二脱氧-D-葡萄糖醇,在Tay-Sachs小鼠中GM2神经节苷脂的CNS储存被抑制。