Since 2020, aggregated from related topics
Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle weakness and degeneration. It primarily affects boys and is caused by a mutation in the gene responsible for producing a protein called dystrophin, which helps maintain the structure of muscle cells. Without dystrophin, muscle cells become damaged and eventually die, leading to muscle weakness and loss of function. DMD typically becomes apparent in early childhood, with symptoms including difficulty walking, frequent falls, and progressive muscle weakness. Over time, individuals with DMD may experience trouble breathing, heart problems, and complications in other organs. Research in Duchenne muscular dystrophy focuses on developing treatments to slow the progression of the disease, improve quality of life, and ultimately find a cure. Current approaches include gene therapy, stem cell transplantation, and pharmacological interventions to address the underlying genetic mutation or support muscle function. Clinical trials are ongoing to evaluate the safety and effectiveness of these treatments in individuals with DMD.