公开(公告)号：EP1007043B9

公开(公告)日：2004-12-22

申请号：EP98901673.8

申请日：1998-01-13

申请人： MONSANTO COMPANY

发明人： PLATT, Frances, M., University of Oxford ,  NEISES, Gabrielle, R. ,  DWEK, Raymond, A. University of Oxford ,  BUTTERS, Terry D. University of Oxford

IPC分类号： A61K31/445

CPC分类号： A61K31/7004 ,  A61K31/445 ,  A61K31/70 ,  A61K31/7012

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中枢神经系统（CNS）参与的溶酶体贮积症患者的方法。 所述方法包括向所述患者施用小但储存抑制有效量的1,5-亚氨基糖的N-烷基衍生物，其中所述烷基含有约2至约8个碳原子且所述1,5-亚氨基糖为 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-亚氨基糖的O-酰化前药。 在说明性实例中，通过给予1,5-（丁基亚氨基）-1,5-二脱氧-D-葡萄糖醇在Tay-Sachs小鼠中抑制GM2神经节苷脂的CNS储存。

公开(公告)号：EP1007043B1

公开(公告)日：2004-06-09

申请号：EP98901673.8

申请日：1998-01-13

申请人： MONSANTO COMPANY

发明人： PLATT, Frances, M., University of Oxford ,  NEISES, Gabrielle, R. ,  DWEK, Raymond, A. University of Oxford ,  BUTTERS, Terry D. University of Oxford

IPC分类号： A61K31/445

CPC分类号： A61K31/7004 ,  A61K31/445 ,  A61K31/70 ,  A61K31/7012

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.