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  • Title: Sanfilippo disease type B: enzyme replacement and metabolic correction in cultured fibroblasts.
    Author: O'Brien JS, Miller AL, Loverde AW, Veath ML.
    Journal: Science; 1973 Aug 24; 181(4101):753-5. PubMed ID: 4269326.
    Abstract:
    alpha-Acetylglucosaminidase, purified from human placent, corrected the defect in mucopolysaccharide degradation when added to culture fibroblasts from patients with Sanfilippo disease type B. A small cellular concentration of enzyme gave a large corrective effect. The half-life of disappearance of enzyme activity was 4 to 7 days.
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