Revolutionizing the Treatment of Duchenne Muscular Dystrophy (DMD): Advancements in Gene Therapy, Exon Skipping, and Sym
Duchenne Muscular Dystrophy (DMD) is a severe X-linked genetic disorder caused by mutations in the dystrophin gene, leading to progressive muscle degeneration and weakness, primarily affecting boys. Early symptoms often include difficulty walking, with patients eventually requiring wheelchair assistance and respiratory support as the disease advances to affect cardiac and respiratory muscles.
Treatment has significantly evolved beyond traditional corticosteroids, incorporating groundbreaking techniques like exon-skipping drugs to partially restore the dystrophin protein and various forms of gene therapy. These targeted approaches aim to slow disease progression, improve muscle function, and extend the quality and duration of life for affected individuals by addressing the underlying genetic defect.