Duchene Muscular Dystrophy

Duchenne Muscular Dystrophy (DMD) is a rare, progressive muscle disorder caused by a mutation in the gene encoding the protein dystrophin. Symptoms typically begin to manifest in early childhood, including skeletal and muscle weakness, leading to limited mobility, reduced muscle tone and size, and an increased risk of cardiomyopathy and respiratory failure. DMD has no cure and treatments are limited to managing symptoms. Physical therapy, steroids and gene therapy show promise in mitigating disease severity and improving overall quality of life. DMD remains an area of active research, with the goal of developing more effective treatments and ultimately a cure.