What is Duchenne Muscular Dystrophy (DMD)?
Duchenne Muscular Dystrophy (DMD) is a fatal genetic disorder characterized by the progressive loss of muscle strength and function. Mostly boys are the victims of DMD. The disease occurs in approximately 1 in every 3,500 boys. Genetic mutations that result in the absence or a defect in dystrophin is found to be the cause of DMD. Dystrophin is a protein necessary to maintain the structural integrity of muscle fibres. This results in a progressive decline of muscle strength. Gradually, the patients with DMD lose their ability to walk, breathe and live independently. Acceleron is a privately held biopharmaceutical company which develops biotherapeutics that modulate the growth of red blood cells, bone, muscle, fat and the vasculature to treat musculoskeletal, metabolic and cancer-related diseases.
Duchenne muscular dystrophy (DMD) is the most well-known of the nine types of muscular dystrophy. It is a genetic disease passed from one generation to the next. Females carry the defective gene that causes the disorder, but males almost exclusively are affected by the disease. When a mother carries the defective gene, her female children have a fifty percent chance of carrying the gene also, but they will not show any symptoms. However, each male child has a fifty percent chance of having the disease and showing signs of the disorder. Although the disease is present from conception, symptoms usually do not develop until the child is five or six years old, or even a year or two later. What are some signs and symptoms of DMD? • With rare exceptions, DMD affects only males. • Unless a boy with DMD is known to be at risk because of his family history, he is unlikely to be diagnosed before the age of 2 or 3 years or older. • Most boys with DMD walk alone at a later age than average, and ev
