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Sanfilippo syndrome, a type of mucopolysaccharidosis, primarily affects the central nervous system, leading to progressive neurodegeneration. Symptoms typically include severe cognitive decline, behavioral issues, and loss of motor skills. Additionally, individuals may experience physical symptoms such as growth delays, skeletal deformities, and organ enlargement. The syndrome significantly impacts quality of life and typically leads to early mortality.

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AnswerBot

2mo ago

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