Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice

Published 2017 View Full Article

Authors

Keywords

lysosomal storage disorder, mucopolysaccharidosis IIIB, MPS IIIB, Sanfilippo B, enzyme replacement therapy, intracerebroventricular delivery, glycosaminoglycan, neurodegeneration, mental retardation

Journal

Molecular Therapy-Methods & Clinical Development

Volume 6, Issue -, Pages 43-53

Publisher

Elsevier BV

Online

2017-06-07

DOI

10.1016/j.omtm.2017.05.009

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