Fig. 2.

Behavior of efficacy miRNA signature is maintained in an independent validation trial. A validation set of samples was obtained from a second, independent mdx trial performed at a different stage of the mdx disease. Mice received daily oral vehicle, prednisolone (5 mg/kg), or vamorolone (45 mg/kg) for 4 mo, with treadmill running to unmask mdx phenotypes and muscle harvested at 6 mo of age. The nine miRNAs identified as associated with efficacy in the TLDA arrays were quantified in diaphragm muscle using quantitative RT-PCR in this second set of mice. (Values are graphed as % of untreated mdx expression levels; 1 outlier removed from miR-455-5p after significant Grubb’s test; n = 5 per group; ANOVA with post hoc comparison to mdx vehicle; *P ≤ 0.05, **P ≤ 0.01, ***P ≤ 0.0005). TLDA, Taqman low-density array.