Duchenne Muscular Dystrophy

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“Duchenne Muscular Dystrophy”

Muscular Dystrophy (MD) is a group of diseases characterized by progressive weakness and degeneration. The skeletal or voluntary muscles are also affected in some forms of MD. A few also include organs too. One of the major forms is Duchenne. Duchenne is the most common form of MD which affect’s children. There is no treatment for any type of MD. Physical Therapy would probably be the only type of treatment available today. People with MD use physical therapy to prevent contractors (a condition in which shortened muscles around the joints cause abnormal and sometimes painful positioning of the joints). Corrective Orthopedic surgery may be needed to improve the quality of life in some cases. The prognoses of MD depends on the type of MD and the progression of the disorder. For instance, in some cases, many are mild and slowly progressive while others may be severe and rapid in progression. With Duchenne MD, death occurs in the late teens to the early twenty’s. Duchenne Muscular Dystrophy (DMD) is one of a group of muscular dystrophies which is also characterized by the enlargement of muscles. DMD is one of the most prevalent types of Muscular Dystrophies. DMD is a specific “type” known by rapid progression of muscle degenereation which occurs early in life. An estimated 1 in 3,500 boys world wide have DMD. The gene for DMD in on the X chromosome which encodes a large protein - Dystrophin. Dystrophin is necessary inside muscle cells for structural support. It is needed to strengthen muscle cells by anchoring elements of the internal cytoskeleton to the surface of the membrane. Without Dystrophin, the cell membrane becomes permeable, so that extracellular components enter the cell increasing the internal pressure until the muscle cell explodes and dies. The fast response of the immune system can add to the damage.
The mouse model for DMD still exists. It’s proving useful for furthering our ...