Sanfilippo Syndrome A (MPS IIIA)

Mucopolysaccharidosis type IIIA (MPS IIIA) is a genetic disorder that makes the body unable to break down large sugar molecules called glycosaminoglycans (GAGs, formerly called mucopolysaccharides). MPS IIIA is the most severe of the MPS III disorders and children with this condition have the shortest survival rate among those with the MPS III disorders. Affected individuals can have severe neurological symptoms, including progressive dementia, aggressive behavior, hyperactivity, seizures, deafness, loss of vision, and an inability to sleep for more than a few hours at a time. (Source: Genetic and Rare Disease Information Center)

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