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Using a sensitive, two-site enzyme-linked immunoassay, we examined the content of nerve growth factor in the cerebrospinal fluid of 11 children with Rett syndrome and of 24 control patients with various neurologic diagnoses or suffering from other diseases. Nerve growth factor levels were significantly lower in the cerebrospinal fluid of the patients with Rett syndrome than in control patients. The lower level of cerebrospinal fluid nerve growth factor in Rett syndrome suggests that lack of nerve growth factor may be involved in the pathogenesis of this disease or reflect the underlying brain damage present. 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