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filingDate 2013-12-06-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_f59e22de087a56e4c243a2df36e388c8
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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.
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