A phase 1/2 study of intrathecal heparan-N-sulfatase in patients with mucopolysaccharidosis IIIA

Published 2016 View Full Article

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Title
A phase 1/2 study of intrathecal heparan-N-sulfatase in patients with mucopolysaccharidosis IIIA
Authors
Keywords
Enzyme replacement therapy, Heparan sulfate, Intrathecal drug delivery device, Lysosomal storage disease, Mucopolysaccharidosis IIIA (MPS IIIA), Sanfilippo syndrome A
Journal
MOLECULAR GENETICS AND METABOLISM
Volume 118, Issue 3, Pages 198-205
Publisher
Elsevier BV
Online
2016-05-11
DOI
10.1016/j.ymgme.2016.05.006

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