The prospects and challenges of small molecule drugs in the treatment of Duchenne muscular dystrophy.

Duchenne muscular dystrophy (DMD) is an X-linked disorder caused by mutations in the DMD gene, leading to reduced or absent dystrophin expression. This results in progressive muscle degeneration, cardiomyopathy, respiratory failure, and ultimately death. Conventional glucocorticoid treatments slow disease progression but cause severe side effects with long-term use. Currently, no cure exists for DMD, so developing new therapies remains a priority. This review highlighted recent advances in small molecule drug treatments, focusing on their mechanisms of action, binding models and synthetic pathways related to target proteins, providing insights for future research.