Treatment of a mouse model of Sanfilippo syndrome, or mucopolysaccharidosis IIIB, an accumulation of heparin sulfate that results in dementia and other neurological disorders, with an altered alpha-N-acetylglucosaminidase enzyme injected into the brain’s ventricular system, decreased heparin sulfate accumulation to non-pathological levels, and suggests a potential enzyme replacement approach to treating the disease, according to a study.
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