Abstract:subjects could sit unassisted. At 24 months after gene therapy, 64% of subjects achieved head control, 50% could sit unassisted, and 18% could stand with support. At 60 months after gene therapy, 75% of subjects achieved head control, 67% of subjects could sit unassisted, 25% of subjects could stand with support, and 8% subjects could walk with support. In contrast, only 4% of the subjects in the NHDB control group achieved key motor milestones (P<0.0001 ). These results demonstrate that the attainment of mile… Show more
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