As there is a clear relationship between the levels of dystrophin expression and the levels of regeneration in patients with BMD [25, 40] and in view of the findings of decrease degeneration/regeneration in DMD preclinical models [37, 47], we decided to investigate the surrogate molecular functionality of induced dystrophin in humans following a 48-week period of therapeutic intervention with exon 53 skipping PMO golodirsen. The gene discussed is DMD; the disease is Duchenne muscular dystrophy.