Method for treatment of CNS-involved lysosomal storage diseases
    5.
    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。