A possible explanation for the lack of significant difference in the level of miRNAs in pre-treatment and post-treatment samples in patients recruited in our trial is that, although 12 weeks of treatment induced restoration of dystrophin expression in DMD patients [19], the 12-week time point might not be sufficient to assess response to therapy at the miRNA level, as the amount of dystrophin is still slowly increasing. The gene discussed is DMD; the disease is Duchenne muscular dystrophy.