http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2017015375-A1
Outgoing Links
| Predicate | Object |
|---|---|
| assignee | http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_a43ef275ae6f6fafc66975041eb60aa7 |
| classificationCPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12Y310-01001 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K9-0019 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K38-46 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61P3-00 |
| classificationIPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K38-46 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P43-00 |
| filingDate | 2016-07-20-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| inventor | http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_feb675446d8143a109c270aa2b89718a |
| publicationDate | 2017-01-26-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber | WO-2017015375-A1 |
| titleOfInvention | Treatment of cognitive impairment of mucopolysaccharidosis type iiia by intrathecal delivery of heparan n-sulfatase |
| abstract | The present invention provides, among other things, effective treatment for Sanfilippo Syndrome Type A (MPS IIIA) based on intrathecal delivery of recombinant heparan N- sulfatase (HNS) enzyme. The present invention also 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) relative to baseline (e.g., prior to treatment) as well as to improve, stabilize, or reduce decline of cognitive function, disability, behavior, quality of life and/or auditory brainstem response relative to baseline (e.g., prior to treatment). |
| priorityDate | 2015-07-20-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type | http://data.epo.org/linked-data/def/patent/Publication |
Incoming Links
Total number of triples: 192.