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Neurofibromatosis type 1 (NF1) is a relatively common autosomal-dominant genetic disorder (incidence, 1:3000), and, among genetic disorders, it ranks as a top cause of cognitive dysfunction. Because animal models suggest that 3-hydroxy-3-methyl-glutaryl-CoA reductase inhibition protects against cognitive loss, Dutch investigators randomized 62 children (mean age, about 12 years) with NF1 to simvastatin (20–40 mg daily, depending on age) or placebo. Children were assessed at baseline on a wide range of structured cognitive tasks. Mean IQ was in the high 80s, mean total cholesterol level was about 160 mg/dL, and mean LDL cholesterol level was about 100 mg/dL.
After 12 weeks, retesting identified no differences between treated and placebo group…