Spinal muscular atrophy is a disease of progressive muscle wasting and motor weakness. In spinal muscular atrophy, the alpha motor neurons in the anterior horn of the spinal cord that are responsible for skeletal muscle control deteriorate and are lost over time. Affected individuals are plagued by muscle weakness that progresses in a proximal to distal fashion. As the motor neurons deteriorate, muscle wasting ensues. Three major types exist (Table 1) and differ based on age of onset and clinical severity of symptoms.
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