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Common cholesterol drug safe, may improve learning disabilities in patients with neurofibromatosis
Date:9/27/2011

Washington, DCResearchers at Children's National Medical Center have found that a cholesterol-lowering statin drug appears to be safe in children with neurofibromatosis type 1 (NF1) and may improve learning disabilities, including verbal and nonverbal memory. This is the first time that the drug lovastatin has been studied in children with NF1. The study, led by Maria T. Acosta, MD, a pediatric neurologist and researcher at Children's National and clinical director and cognitive director of the Gilbert Family Neurofibromatosis Institute, appears in the October 2011 issue of Pediatric Neurology.

Lovastatin is currently approved by the U.S. Food and Drug Administration in adults and children for the treatment of high cholesterol. The drug works by inhibiting a specific enzyme in cholesterol biosynthesis. Previous animal studies have found that lovastatin affects a related molecular pathway that may be linked to cognitive deficits in neurofibromatosis.

"While we originally set out to determine the safety of lovastatin in NF1 patients, we also saw statistical improvements in memory and visual attention, which is a big step towards helping improve our patients' quality of life and in evaluating biologic agents which may be effective therapies for NF1," stated Dr. Acosta. "While this is a relatively small study, we now have strong baseline information, and we are working with other institutions in the country and throughout t
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Contact: Emily Hartman
ehartman@childrensnational.org
202-476-4500
Children's National Medical Center
Source:Eurekalert

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