Method to predict response to pharmacological chaperone treatment of diseases
US9095584B2 · kind B2 · utility
50Cited by
1References
10Claims
0Family size
Assignee
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Key dates
| Filing date | Oct 15, 2013 |
| Grant date | Aug 4, 2015 |
| Priority date | — |
| Expiry date | Oct 15, 2033 |
Classification
- Technology area (CPC G)Physics
- CPC primaryG01N2800/52
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention provides methods to determine whether a patient with a lysosomal storage disorder win benefit from treatment with a specific pharmacological chaperone. The present invention exemplifies an in vitro method for determining α-galactosidase A responsiveness to a pharmacological chaperone such as 1-deoxygalactonojirimycin in a cell line expressing a mutant from of α-galactosidase A. The invention also provides a method for diagnosing Fabry disease in patients suspected of having Fabry disease.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.