| Predicate |
Object |
| assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_4d5de9a1dac6b033c4643bb236ef4df3 |
| classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K31-445
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K31-49
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K31-4025
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K2300-00 |
| classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P25-16
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K31-443
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P43-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K31-501
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K31-341
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K31-351
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K9-0053
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K31-4155 |
| classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P25-16
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K31-4155
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P43-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K31-443
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K31-341
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K31-501
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K9-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K31-351 |
| filingDate |
2018-07-16-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_04c40dca471945af4b3c090500c66265
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_5dc4357cb2d8f09ae9054438ddc3c8fc |
| publicationDate |
2019-01-17-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber |
US-2019015380-A1 |
| titleOfInvention |
Cns-accessible pharmacological chaperones for treatment of acid beta-glucosidase-related disease states |
| abstract |
Disclosed herein are β-glucosidase (GCase) chaperones and methods of using GCase chaperones in an individual in need thereof. GBA1 mutations lead to GCase deficiency and substrate accumulation, causing Gaucher disease. Currently, no FDA or EMA-approved therapeutic for neuronopathic Gaucher disease is available. Improved GCase activity in brain cells using a chaperone may reduce substrate accumulation and associated pathology. Disclosed herein are novel non-inhibitory chaperone compounds of GCase that have properties of a central nervous system drug. Those compounds effectively restored mutant GCase activity by stabilizing protein and enhancing lysosomal localization and may be useful for chaperone therapy to treat neuronopathic Gaucher disease and likely to Parkinson's disease. |
| isCitedBy |
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/CN-111892511-A
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/CN-114040762-A |
| priorityDate |
2017-07-17-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type |
http://data.epo.org/linked-data/def/patent/Publication |