发明公开
EP2823043A1 HIGH CONCENTRATION ALPHA-GLUCOSIDASE COMPOSITIONS FOR THE TREATMENT OF POMPE DISEASE
审中-公开
用于治疗蓬珀病高浓度α-葡萄糖苷酶的组合物
- 专利标题: HIGH CONCENTRATION ALPHA-GLUCOSIDASE COMPOSITIONS FOR THE TREATMENT OF POMPE DISEASE
- 专利标题(中): 用于治疗蓬珀病高浓度α-葡萄糖苷酶的组合物
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申请号: EP13757576.7申请日: 2013-03-07
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公开(公告)号: EP2823043A1公开(公告)日: 2015-01-14
- 发明人: VALENZANO, Kenneth, Joseph , CROWLEY, John , KHANNA, Richie , FLANAGAN, John
- 申请人: Amicus Therapeutics, Inc.
- 申请人地址: 1 Cedar Brook Drive Cranbury, NJ 08512 US
- 专利权人: Amicus Therapeutics, Inc.
- 当前专利权人: Amicus Therapeutics, Inc.
- 当前专利权人地址: 1 Cedar Brook Drive Cranbury, NJ 08512 US
- 代理机构: Miller Sturt Kenyon
- 优先权: US201261607920P 20120307; US201361750718P 20130109
- 国际公布: WO2013134530 20130912
- 主分类号: C12N9/40
- IPC分类号: C12N9/40 ; C07D211/06 ; A61K31/58 ; C12N9/99
摘要:
The present application provides for compositions comprising high concentrations of acid a-glucosidase in combination with an active site-specific chaperone for the acid α-glucosidase, and methods for treating Pompe disease in a subject in need thereof, that includes a method of administering to the subject such compositions. The present application also provides methods for increasing the in vitro and in vivo stability of an acid α-glucosidase enzyme formulation.
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