Invention Grant
- Patent Title: Treatment of Fabry disease
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Application No.: US16313057Application Date: 2017-06-14
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Publication No.: US10995067B2Publication Date: 2021-05-04
- Inventor: Wei-Chieh Cheng
- Applicant: Academia Sinica
- Applicant Address: TW Taipei
- Assignee: Academia Sinica
- Current Assignee: Academia Sinica
- Current Assignee Address: TW Taipei
- International Application: PCT/US2017/037381 WO 20170614
- International Announcement: WO2017/222881 WO 20171228
- Main IPC: A61K31/7028
- IPC: A61K31/7028 ; C07D207/02 ; C07D207/12 ; A61K31/40 ; A61K45/06 ; A61P3/00 ; A61K38/47

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).
Public/Granted literature
- US20190225579A1 TREATMENT OF FABRY DISEASE Public/Granted day:2019-07-25
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