Fig. 4

Change in NSAA score at week 24. Change in NSAA score from baseline to after 24 weeks of treatment. In general, a higher score indicates better motor function. For the mean difference, gain of score is shown in negative numbers and loss of score in positive numbers in the figure, i.e., 4.15 indicates a negative score of − 4.15 in the actual measurement. As the trial by Voit2014 had only provided the adjusted mean difference versus placebo, SDs for each intervention group could not be calculated and were substituted with SDs reported in the NCT01462292 trial. There was no significant change in the NSAA scores between the treated and placebo groups. Subgroup analysis revealed a significant increase in the scores in the placebo groups compared with those in the eteplirsen group