发明名称 |
A METHOD FOR THE TREATMENT OF POMPE DISEASE USING 1- DEOXYNOJIRIMYCIN DERIVATIVES |
摘要 |
<p>The present invention provides a method for increasing the activity of a mutant or wild-type alpha-glucosidase enzyme in vitro and in vivo by contacting the enzyme with a specific pharmacological chaperone which is a derivative of 1-deoxynojirimycin. The invention also provides a method for the treatment of Pompe disease by administration of chaperone small molecule compound which is a derivative of 1-deoxynojirimycin. The 1-deoxynojirimycin derivative is substituted at the N or C1 position. Combination therapy with replacement alpha-glucosidase gene or enzyme is also provided.</p> |
申请公布号 |
IL209951(D0) |
申请公布日期 |
2011.02.28 |
申请号 |
IL20100209951 |
申请日期 |
2010.12.13 |
申请人 |
AMICUS THERAPEUTICS, INC. |
发明人 |
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分类号 |
A61K |
主分类号 |
A61K |
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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