Ambroxol for the treatment of patients with Parkinson disease with and without glucocerebrosidase gene mutations: a nonrandomized, noncontrolled trial
S Mullin, L Smith, K Lee, G D'Souza… - JAMA …, 2020 - jamanetwork.com
Importance Mutations of the glucocerebrosidase gene, GBA1 (OMIM606463), 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 assess safety, tolerability,
cerebrospinal fluid (CSF) penetration, and target engagement of ambroxol therapy with …
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 assess safety, tolerability,
cerebrospinal fluid (CSF) penetration, and target engagement of ambroxol therapy with …
