Treatment of cognitive impairment of mucopolysaccharidosis type IIIA by intrathecal delivery of heparan N-sulfatase
US10639356B2 · kind B2 · utility
Assignee
Inventor
Key dates
| Filing date | Jul 20, 2016 |
| Grant date | May 5, 2020 |
| Priority date | — |
| Expiry date | Aug 8, 2036 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12Y310/01001
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
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).
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.