Representative neurologic function of treated and untreated Sandhoff mice.

Each animal was tested for its ability to cross a horizontal bar, measuring balance and motor co-ordination (A), and for the amount of time it spent upside down on an inverted screen, measuring muscle strength (B). Data are mean ± 0.4 of the SD. n = 4 to 6 animals per group. Locomotor activity of the NB-DNJ–treated and untreated mice was measured (C). Each animal at 12, 18, and 24 weeks of age was tested for locomotion in an “open-field.” Data are mean ± SD (n = 4-6 animals per group). Between treated and untreated mice, data are statistically significant at 18 weeks (P < .05) and 24 weeks (P < .01) of age in control and BMT-treated Sandhoff mice.