http://rdf.ncbi.nlm.nih.gov/pubchem/patent/EP-3471736-A1
Outgoing Links
Predicate | Object |
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assignee | http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_8bd7a45a343acd10e3f5d8775dda2157 |
classificationCPCAdditional | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12Y302-01022 |
classificationCPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07D207-12 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K31-7028 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P3-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07D207-02 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K45-06 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K38-47 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K31-40 |
classificationIPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C07D207-02 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K31-7028 |
filingDate | 2017-06-14-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
inventor | http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_4dd46f244c25cee46c5dfc0681887272 |
publicationDate | 2019-04-24-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
publicationNumber | EP-3471736-A1 |
titleOfInvention | Treatment of fabry disease |
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: R 1 is H, or C 1-3 amine optionally substituted with -COR 2 ; R 2 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). |
priorityDate | 2016-06-24-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
type | http://data.epo.org/linked-data/def/patent/Publication |
Incoming Links
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