TY - JOUR AU - Silveira, C. R. A. AU - MacKinley, J. AU - Coleman, K. AU - Li, Z. AU - Finger, E. AU - Bartha, R. AU - Morrow, S. A. AU - Wells, J. AU - Borrie, M. AU - Tirona, R. G. AU - Rupar, C. A. AU - Zou, G. AU - Hegele, R. A. AU - Mahuran, D. AU - MacDonald, P. AU - Jenkins, M. E. AU - Jog, M. AU - Pasternak, S. H. PY - 2019 DA - 2019/02/09 TI - Ambroxol as a novel disease-modifying treatment for Parkinson’s disease dementia: protocol for a single-centre, randomized, double-blind, placebo-controlled trial JO - BMC Neurology SP - 20 VL - 19 IS - 1 AB - Currently there are no disease-modifying treatments for Parkinson’s disease dementia (PDD), a condition linked to aggregation of the protein α-synuclein in subcortical and cortical brain areas. One of the leading genetic risk factors for Parkinson's disease is being a carrier in the gene for β-Glucocerebrosidase (GCase; gene name GBA1). Studies in cell culture and animal models have shown that raising the levels of GCase can decrease levels of α-synuclein. Ambroxol is a pharmacological chaperone for GCase and is able to raise the levels of GCase and could therefore be a disease-modifying treatment for PDD. The aims of this trial are to determine if Ambroxol is safe and well-tolerated by individuals with PDD and if Ambroxol affects cognitive, biochemical, and neuroimaging measures. SN - 1471-2377 UR - https://doi.org/10.1186/s12883-019-1252-3 DO - 10.1186/s12883-019-1252-3 ID - Silveira2019 ER -