A phase 1/2 study of intrathecal heparan-N-sulfatase in patients with mucopolysaccharidosis IIIA
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open access
Abbreviations
AE
adverse event
BSID-III
Bayley Scales of Infant and Toddler Development, Third Edition
CSF
cerebrospinal fluid
CNS
central nervous system
DQ
developmental quotient
EC
Enzyme Commission
ELISA
enzyme-linked immunosorbent assay
GAG
glycosaminoglycan
IDDD
intrathecal drug delivery device
IT
intrathecal
KABC-II
Kaufman Assessment Battery for Children, Second Edition
LC-MS/MS
liquid chromatography-tandem mass spectrometry
MPS IIIA
mucopolysaccharidosis IIIA/Sanfilippo syndrome type A
MRI
magnetic resonance imaging
rhHNS
recombinant human heparan-N-sulfatase (EC 3.10.1.1)
SAE
serious adverse event
TEAE
treatment-emergent adverse event
Tmax
time to maximum serum concentration
uGAG
urinary glycosaminoglycan
VABS-II
Vineland Adaptive Behavior Scales, Second Edition
Keywords
Enzyme replacement therapy
Heparan sulfate
Intrathecal drug delivery device
Lysosomal storage disease
Mucopolysaccharidosis IIIA (MPS IIIA)
Sanfilippo syndrome A
Cited by (0)
- 1
Current address: Vertex Pharmaceuticals, Inc., 50 Northern Ave, Boston, MA 02210.
- 2
Current address: Alnylam Pharmaceuticals, Inc., 300 Third Street, Cambridge, MA 02142.
- 3
Current address: Agios Pharmaceuticals, 88 Sidney Street, Cambridge, MA 02139.
© 2016 The Authors. Published by Elsevier Inc.