Method to predict response to pharmacological chaperone treatment of diseases
USRE48608E1 · kind E1 · reissue
35Cited by
40References
44Claims
0Family size
Assignee
Inventors
Key dates
| Filing date | Dec 17, 2018 |
| Grant date | Jun 29, 2021 |
| Priority date | — |
| Expiry date | Dec 17, 2038 |
Classification
- Technology area (CPC —)General
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.