http://rdf.ncbi.nlm.nih.gov/pubchem/patent/EP-3471736-A1

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classificationCPCAdditional http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12Y302-01022
classificationCPCInventive http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C07D207-12
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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

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http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ0TKT1
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP77989
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCA0KQH4
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ7TQD2
http://rdf.ncbi.nlm.nih.gov/pubchem/taxonomy/TAXID54755
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID45136662
http://rdf.ncbi.nlm.nih.gov/pubchem/substance/SID419520697
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCA4TLL5
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ42656
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID42796
http://rdf.ncbi.nlm.nih.gov/pubchem/substance/SID226394131
http://rdf.ncbi.nlm.nih.gov/pubchem/compound/CID444913
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCO52847
http://rdf.ncbi.nlm.nih.gov/pubchem/substance/SID421537451
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ8A040

Total number of triples: 324.