Rodent Duchenne Muscular Dystrophy (DMD) Model

DMD is a progressive muscle wasting disease that affects approximately 1 in 3500 male births. It is caused by mutations in a large 79-exon gene encoding the dystrophin protein. The majority of mutations identified in DMD are frameshift mutations that result in complete loss of dystrophin. Patients typically present at 2-5 years of age with a waddling gait, calf hypertrophy, and difficulties in rising from the floor.There is currently no cure for DMD and clinical management involves treatment with steroids combined with respiratory and cardiac support. Preclinical DMD research has benefited from the availability of a number of animal models of the disease, among which the mdx mouse is the most commonly used and best characterized.