Figure 3.

Reduction of Lysosomal Storage and Elevated β-hexosaminidase Activity by BMN 250 at 1 and 28 Days following Four 100 μg ICV Doses over 2 Weeks

(A) Steady-state levels for total HS and disease-specific NREs in brain homogenate, shown as picomoles per milligram of tissue wet weight (mean ± SEM). (B) β-Hexosaminidase enzyme activity (mean ± SEM) measured in brain homogenate. VEH, vehicle. n = 6 per group, except n = 2 for naive Naglu^+/− mice. **p < 0.01, ***p < 0.001, ****p < 0.0001; ns, not significant.