Treatment of Fabry disease
US10995067B2 · kind B2 · utility
Assignee
Inventor
Key dates
| Filing date | Jun 14, 2017 |
| Grant date | May 4, 2021 |
| Priority date | — |
| Expiry date | Jun 14, 2037 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12Y302/01022
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Disclosed herein are novel uses of a polyhydroxylated pyrrolidine for the manufacture of a medicament for treating Fabry disease (FD). Accordingly, the present disclosure provides a method of treating a subject having or suspected of having FD. The method includes the step of, administering to the subject a therapeutically effective amount of a compound of formula (I), a salt, an ester or a solvate thereof, wherein: R1 is H, or C1-3 amine optionally substituted with —COR2; R2 is alkyl or alkene optionally substituted with cycloalkyl or phenyl having at least one substituent selected from the group consisting of, halo, alkyl, haloalkyl, and alkoxyl; so as to ameliorate, alleviate mitigate and/or prevent symptoms associated with the FD. According to preferred embodiments of the present disclosure, the compound of formula (I) is a chaperon of a mutated human lysosomal α-galactosidase A (α-Gal A).
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.