Effect of simvastatin on cognitive functioning in children with neurofibromatosis type 1: a randomized controlled trial

LC Krab, A de Goede-Bolder, FK Aarsen, SMF Pluijm… - Jama, 2008 - jamanetwork.com
Context Neurofibromatosis type 1 (NF1) is among the most common genetic disorders that
cause learning disabilities. Recently, it was shown that statin-mediated inhibition of 3-
hydroxy-3-methylglutaryl coenzyme A reductase restores the cognitive deficits in an NF1
mouse model. Objective To determine the effect of simvastatin on neuropsychological,
neurophysiological, and neuroradiological outcome measures in children with NF1. Design,
Setting, and Participants Sixty-two of 114 eligible children (54%) with NF1 participated in a …