The significance of this event in DM1 pathogenesis was demonstrated in preclinical studies showing that the correction of GSK3β using small-molecule GSK3β inhibitors had positive effects on the improvement of skeletal muscle and CNS defects in DM1 mouse models [20,21,22] and in a human study, a Phase II clinical trial for juvenile and adolescent patients with CDM1 [15,23]. The gene discussed is GSK3B; the disease is myotonic dystrophy type 1.