Method for the treatment of Pompe disease using 1-deoxynojirimycin derivatives
US11602528B2 · kind B2 · utility
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Key dates
| Filing date | Dec 12, 2017 |
| Grant date | Mar 14, 2023 |
| Priority date | — |
| Expiry date | Dec 22, 2038 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC07D409/06
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention provides a method for increasing the activity of a mutant or wild-type α-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 α-glucosidase gene or enzyme is also provided.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.