Evolving Therapeutic Strategies for Duchenne Muscular Dystrophy: Advancing from Supportive Care
Duchenne Muscular Dystrophy (**DMD**) is a severe, rapidly progressing genetic condition resulting from a mutation in the *DMD* gene, leading to the absence of the essential protein **dystrophin**, which is crucial for stabilizing muscle cell membranes during contraction. Without functional dystrophin, muscle tissue undergoes relentless degeneration, inflammation, and eventual replacement by scar tissue and fat, leading to loss of ambulation and severe cardiac and respiratory compromise. The therapeutic approach has undergone a significant transformation, moving from traditional supportive care to complex molecular and genetic strategies aimed at addressing the root cause.
The foundation of care for DMD involves **corticosteroid therapy** (e.g., prednisone or deflazacort), which has been shown to prolong the period of ambulation, stabilize respiratory function, and slow the overall progression of muscle weakening. However, corticosteroids do not cure the underlying condition and are associated with systemic side effects. Simultaneously, supportive management, including physical therapy, orthopedic interventions, and early…