发明名称 |
METHOD FOR THE TREATMENT OF FABRY DISEASE USING PHARMACOLOGICAL CHAPERONES |
摘要 |
The present invention provides a method treating a patient with Fabry disease by determining whether there is an improvement of a surrogate marker that is associated with Fabry disease following administration of a specific pharmacological chaperone of a-galactosidase A. The method includes administering an effective amount of 1-deoxygalactonojirimycn to the individual, wherein the 1- deoxygalactonojirimycin binds to alpha-galactosidase A in an amount effective to increase activity of the alpha-galactosidase A. The present invention also provides a method for monitoring and increasing a therapeutic response of a patient with Fabry disease following administration of a specific pharmacological chaperone of a- galactosidase A by evaluating the effect on the cytoplasmic staining pattern of a cell from the patient, wherein detection of a staining pattern in the cell that is similar to the staining pattern in a cell from a healthy individual indicates that the individual with Fabry disease is a responder. |
申请公布号 |
WO2008121826(A3) |
申请公布日期 |
2008.11.27 |
申请号 |
WO2008US58668 |
申请日期 |
2008.03.28 |
申请人 |
AMICUS THERAPEUTICS, INC.;PALLING, DAVID |
发明人 |
PALLING, DAVID |
分类号 |
A61K33/24 |
主分类号 |
A61K33/24 |
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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