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治療法布瑞氏症
专利权人:
ACADEMIA SINICA
发明人:
CHENG, WEI-CHIEH,郑伟杰,鄭偉杰
申请号:
TW106119737
公开号:
TW201800391A
申请日:
2017.06.14
申请国别(地区):
TW
年份:
2018
代理人:
摘要:
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: R1is H, or C1-3amine optionally substituted with –COR2; R2is 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).本揭示內容是關於一種多羥化吡咯啶的新穎用途,其可用以製備一藥物以治療法布瑞氏症。據此,本揭示內容提供一種用以治療罹患或疑似罹患法布瑞氏症之個體的方法。該方法包含對該個體投予一治療有效量之式(I)化合物、其鹽類、酯類或溶劑合物,其中,R1是H或以–COR2任選取代的C1-3胺;R2是以具有至少一取代基之環烷基或苯基任選取代的烷基或烯烴,其中該取代基是選自由鹵素、烷基、鹵烷基及烷氧基所組成的群組;藉以改善、減緩、減輕及/或預防與法布瑞氏症相關之病徵。依據本揭示內容較佳的實施方式,式(I)化合物為一人類突變溶酶體α-半乳糖苷酶A的伴護蛋白。
来源网站:
中国工程科技知识中心
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