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Ambroxol as a novel disease-modifying treatment for Parkinson's disease dementia: protocol for a single-centre, randomized, double-blind, placebo-controlled trial.

著者 Silveira CRA , MacKinley J , Coleman K , Li Z , Finger E , Bartha R , Morrow SA , Wells J , Borrie M , Tirona RG , Rupar CA , Zou G , Hegele RA , Mahuran D , MacDonald P , Jenkins ME , Jog M , Pasternak SH
BMC Neurol.2019 Feb 09 ; 19(1):20.
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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.
PMID: 30738426 [PubMed - in process]
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