METHODS AND COMPOSITIONS FOR INTRATHECALLY ADMINISTERED TREATMENT OF MUCUPOLYSACCHARIDOSIS TYPE IIIA
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Dec 6, 2013
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Dec 7, 2012
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Abstract
The present invention provides, among other things, effective treatment for Sanfilippo Syndrome Type A (MPS IIIA) 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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