Intravenous delivery of a chemically modified sulfamidase efficiently reduces heparan sulfate storage and brain pathology in mucopolysaccharidosis IIIA mice.

MPS IIIA mice presented weak memory deficits that improved after CM-rhSulfamidase treatment, although the difference failed to reach statistical significance when compared to vehicle treated controls ( Fig. 10 D). 3.8.

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Trends towards increased activity did not reach statistical significance after correction for group comparisons for the other parameters evaluated ( Fig. 10 A), or at lower doses (data not shown).

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