Fig. 2

5’−3’ DMD transcript imbalance visualization and its restoration after golodirsen treatment. A Boxplot with individual data points offer the visual representation of the DMD transcript imbalance, through transcript length (5’−3’). Transcripts’ expression, calculated by the 2^-DDCt methods from FluiDMD cards data, (Y-axis) were plotted against the average of around 10 exon-exon junctions (X-axis) until the exon-exon junction ex61-ex62, to avoid the Dp71 isoform contribution. Dots indicate values for each individual data point, representing the single patient contribution for each interval of exon-exon junctions. 5’ junctions are higher expressed than 3’ junctions in both treated (pink boxes) and non-treated (blue boxes) groups. Exon-exon junctions surrounding the deletions and downstream the exon 62 were removed to avoid underestimation and the Dp71 contribution, respectively. (Wilcoxon test p-value, *= p < 0.001, **= p < 0.001, ***= p = 0.003. B Transcript imbalance restoration (Y-axis), after golodirsen administration, was evaluated by subtracting from the slope angle value of the treated sample (+ AON) the non-treated sample (-AON) for each patient (X-axis). Only 5 patients did not show any reduction in transcript imbalance after golodirsen (negative bars)