Individual values for participants in the low-dose (filled circles, n = 3) and high-dose (open circles, n = 16) fordadistrogene movaparvovec (FM) groups of the current study are shown along with box and whisker plots. The box plots display the interquartile range from the 25th to the 75th percentile. The line inside the box indicates the median value. The whiskers indicate the range within 1.5 times the interquartile range, with outliers shown as individual points. The small black dot (bottom left) represents an outlier in the external control cohort. The NSAA total score is based on assessment of 17 ambulatory actions and ranges from 0 to 34, with a higher score indicating greater functional ability. In the external control cohort ( n = 59), the median NSAA total score at baseline was 28.0 in the 6–7-year-old group ( n = 23), 28.0 in the 8-year-old group ( n = 15), 26.0 in the 9-year-old group ( n = 11) and 24.5 in the 10–12-year-old group ( n = 10).
