DMD and Duchenne muscular dystrophy: In June 2023, Sarepta Therapeutics’ Elevidys (SRP-9001; delandistrogene moxeparvovec) received accelerated United States (US) Food and Drug Administration (FDA) approval as the first gene therapy/micro-dystrophin treatment for DMD in children aged 4–5 years (Konieczny et al., 2013).