Method to predict response to pharmacological chaperone treatment of diseases
US10813921B2 · kind B2 · utility
37Cited by
5References
18Claims
0Family size
Assignee
Inventors
Key dates
| Filing date | Jul 26, 2018 |
| Grant date | Oct 27, 2020 |
| Priority date | — |
| Expiry date | Jul 26, 2038 |
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 will 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.