Predicate |
Object |
assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_a43ef275ae6f6fafc66975041eb60aa7 |
classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12Y301-06013 |
classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P43-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K9-0019 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K9-0085 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K38-465 |
classificationIPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K38-46 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P43-00 |
filingDate |
2013-12-06-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_f59e22de087a56e4c243a2df36e388c8 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_63d308747196a196ad59195928749d72 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_248b7f93cb750712af3b8c47bd296045 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_9ba43ad568f708b0ad3b1b6a6d54ae97 |
publicationDate |
2014-06-12-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
publicationNumber |
WO-2014089487-A1 |
titleOfInvention |
Methods and compositions for intrathecally administered treatment of mucupolysaccharidosis type iiia |
abstract |
The present invention provides, among other things, effective treatment for Sanfilippo Syndrome Type A (MPS IDA) based on intrathecal delivery of recombinant heparin N-Sulfatase (HNS) enzyme. In some embodiments, the present invention includes methods of treating Sanfilippo Syndrome Type A (MPS IIIA) Syndrome by intrathecal administration of a recombinant HNS enzyme at a therapeutically effective dose and an administration interval for a period sufficient to decrease glycosaminoglycan (GAG) heparan sulfate level in the cerebrospinal fluid (CSF) and/or urine relative to a control. |
isCitedBy |
http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-10213485-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/KR-20170054465-A http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2017015375-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2019145500-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/EP-3827839-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-10702582-B2 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/KR-101950916-B1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-2017333527-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-2019117732-A1 http://rdf.ncbi.nlm.nih.gov/pubchem/patent/US-10639356-B2 |
priorityDate |
2012-12-07-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
type |
http://data.epo.org/linked-data/def/patent/Publication |