Article
Ambroxol for the Treatment of Patients With Parkinson Disease With and Without Glucocerebrosidase Gene Mutations: A Nonrandomized, Noncontrolled Trial.
JAMA neurology - 1 Apr 2020
Mullin Stephen, Smith Laura, Lee Katherine, D'Souza Gayle, Woodgate Philip, Elflein Josh, Hällqvist Jenny, Toffoli Marco, Streeter Adam, Hosking Joanne, Heywood Wendy E, Khengar Rajeshree, Campbell Philip, Hehir Jason, Cable Sarah, Mills Kevin, Zetterberg Henrik, Limousin Patricia, Libri Vincenzo, Foltynie Tom, Schapira Anthony H V
Abstract excerpt
Importance: Mutations of the glucocerebrosidase gene, GBA1 (OMIM 606463), are the most important risk factor for Parkinson disease (PD). In vitro and in vivo studies have reported that ambroxol increases β-glucocerebrosidase (GCase) enzyme activity and reduces α-synuclein levels. These observations support a potential role for ambroxol therapy in modifying a relevant pathogenetic pathway in PD. Objective: To...
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