Pharmacotherapy of Duchenne Muscular Dystrophy (DMD)

Abstract

Duchenne Muscular Dystrophy (DMD) is a severe, progressive neuromuscular disorder caused by mutations in the DMD gene, leading to the absence of functional dystrophin protein. Over time, it results in muscle weakness and degeneration, causing loss of mobility, respiratory failure, and ultimately, death. This necessitates the need for effective pharmacotherapy to slow the disease progression and improve patient outcomes. The review discusses corticosteroids, which are the gold standard for treating the downstream pathological changes seen in DMD. Novel therapies such as exon skipping, stop codon readthrough, and gene therapies have proven to be reliable in treating some, if not all, mutation variants. With advancements in medical research and technologies, we can hope for better disease management and extended life expectancy.