DMD and Duchenne muscular dystrophy: In 2019, the Food and Drug Administration (FDA) gave accelerated approval to golodirsen for the treatment of DMD patients with eligible deletions, based on the increases of dystrophin expression observed in the clinical study SRP-4053, in which golodirsen resulted in an unequivocal increase in protein expression confirmed with western blot and semiquantitative immunohistochemistry [14], despite a degree of inter-patient variability [23].