Abstract

Background and Objectives: Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disorder caused by DMD pathogenic variants, leading to dystrophin deficiency, muscle degeneration, loss of ambulation, respiratory failure, and reduced life expectancy. Current treatments, such as corticosteroids and supportive care, offer limited long-term benefits. Delandistrogene moxeparvovec is a promising gene therapy developed to restore dystrophin expression and deliver...