Title : Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome.

Pub. Date : 2021 Jun

PMID : 33839004






1 Functional Relationships(s)
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1 Mucopolysaccharidosis IIIB (MPS IIIB, Sanfilippo syndrome type B) is caused by a deficiency in alpha-N-acetylglucosaminidase (NAGLU) activity, which leads to the accumulation of heparan sulfate (HS). Heparitin Sulfate N-acetyl-alpha-glucosaminidase Homo sapiens