Method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
US9181184B2 · kind B2 · utility
25Cited by
15References
4Claims
0Family size
Assignee
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Key dates
| Filing date | May 17, 2006 |
| Grant date | Nov 10, 2015 |
| Priority date | — |
| Expiry date | Apr 21, 2029 |
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.