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. 2020 Apr 8;28(6):1442–1454. doi: 10.1016/j.ymthe.2020.03.018

Figure 2.

Figure 2

The Efficacy of PS822 in Neonatal MPS I Mice

(A) Blood samples were collected from treated neonatal mice and controls monthly. Plasma IDUA enzyme activity increased significantly throughout 10 months. (B) Kaplan-Meier analysis showed the improved survival rate of treated neonatal mice. Three mice from the high-dose group were euthanized at 1 month post-dosing for early assessment and thus were not included in the Kaplan-Meier survival analysis. p < 0.05 when comparing each treatment group to untreated MPS I mice (log rank [Mantel-Cox] test for survival analysis. (C) Fear conditioning showed treated neonatal mice had better memory and learning ability. Mean ± SEM. ∗p < 0.05 when comparing treated to untreated MPS I mice; ∗∗p < 0.01. One-way ANOVA for multiple comparisons).