Despite the cause of DMD being attributed to mutations in dystrophin after identification of the DMD gene in 1987 (Hoffman et al., 1987), treatment options have been limited to corticosteroid therapy via administration of prednisone/prednisolone, deflazacort [approved by the US Food and Drug Administration (FDA) in 2017] and, more recently, vamorolone (AGAMREE; approved by the FDA and European Medicines Agency in 2023). Here, DMD is linked to Duchenne muscular dystrophy.