TY - JOUR AU - Korf, Bruce R. PY - 2008 DA - 2008/07/31 TI - Statins, bone, and neurofibromatosis type 1 JO - BMC Medicine SP - 22 VL - 6 IS - 1 AB - Neurofibromatosis type 1 (NF1) is a dominantly inherited multi-system disorder. Major features include pigmentary abnormalities, benign tumors of the nerve sheath (neurofibromas), malignant tumors, learning disabilities, and skeletal dysplasia. The NF1 gene functions as a tumor suppressor, but haploinsuffiency probably accounts for some aspects of the non-tumor phenotype. The protein product, neurofibromin, is a Ras GTPase-activating protein, and various Ras pathway inhibitors are being tested in preclinical models and clinical trials for effectiveness in treating NF1 complications. This month in BMC Medicine, a paper by Kolanczyk et al describes a preclinical mouse model for tibial dysplasia and provides evidence that the drug lovastatin – in use to treat cardiovascular disease – may be beneficial, opening the door to clinical trials in humans. SN - 1741-7015 UR - https://doi.org/10.1186/1741-7015-6-22 DO - 10.1186/1741-7015-6-22 ID - Korf2008 ER -