Abstract

Duchenne muscular dystrophy (DMD) is a severe, hereditary neuromuscular disorder characterized by the absence of the functional dystrophin protein. This deficiency leaves muscle fibers highly vulnerable to contraction-induced damage, triggering a cycle of chronic inflammation and the unregulated deposition of extracellular matrix (ECM). As the disease progresses, healthy tissue is replaced by dense fibrotic scarring, which leads to a loss of mobility and fatal respiratory or cardiac complications. Driven by the critical need to address this pathology, the global DMD treatment market is expanding rapidly, transitioning from traditional symptom-management strategies, such as corticosteroids and physical rehabilitation, to targeted, molecular-based interventions like exon-skipping and gene replacement therapies. Ultimately, integrating advanced biotechnologies, localized delivery mechanisms, and ongoing supportive care will be vital to actively regenerate functional skeletal muscle and extend the quality of life for patients living with DMD.

Keywords

Duchenne muscular dystrophy, dystrophin restoration, cell-based therapies, biomaterial scaffolds, tissue engineering, regenerative medicine