发明名称 |
HIGH CONCENTRATION ALPHA-GLUCOSIDASE COMPOSITIONS FOR THE TREATMENT OF POMPE DISEASE |
摘要 |
The present application provides for compositions comprising high concentrations of acid α-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. |
申请公布号 |
EP2823043(A1) |
申请公布日期 |
2015.01.14 |
申请号 |
EP20130757576 |
申请日期 |
2013.03.07 |
申请人 |
AMICUS THERAPEUTICS, INC. |
发明人 |
VALENZANO, KENNETH, JOSEPH;CROWLEY, JOHN;KHANNA, RICHIE;FLANAGAN, JOHN |
分类号 |
A61K31/58;A61K38/47;C12N9/99 |
主分类号 |
A61K31/58 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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