DMD and Duchenne muscular dystrophy: Many therapeutic approaches for DMD are in development, aiming to restore dystrophin (e.g. gene replacement with microdystrophin, exon skipping, gene editing, stop-codon readthrough) or upregulate utrophin, or targeting downstream effects of dystrophin deficiency to preserve muscle tissue for as long as possible (reviewed by Duan et al., 2021; Kupatt et al., 2021).