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November 26, 2008

Statin Therapy and Cognitive Deficits Associated With Neurofibromatosis Type 1

JAMA. 2008;300(20):2369-2370. doi:10.1001/jama.2008.687

To the Editor: Dr Krab and colleagues1 investigated the possible benefit of statin therapy in children with neurofibromatosis type 1 (NF1), a genetic disorder associated with learning disabilities.2 In this randomized, double-blind, placebo-controlled trial, it was found that simvastatin, a 3-hydroxy-3-methylglutaryl coenzyme A (HMG-CoA) reductase inhibitor, did not improve cognitive function in children with NF1.1 The motivation for this clinical trial came from the beneficial effect of lovastatin (another HMG-CoA reductase inhibitor) on cognitive function in the Nf1+/− mouse model NF1.3 These mice are heterozygous for a null mutation in neurofibromin, exhibit behavioral disorders that resemble those found in humans, and display deficits in physiological correlates of memory.3