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Ambroxol for the Treatment of Patients With Parkinson Disease With and Without Glucocerebrosidase Gene Mutations: A Nonrandomized, Noncontrolled Trial

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...

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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Medical Association 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6990847/
https://www.ncbi.nlm.nih.gov/pubmed/31930374
http://dx.doi.org/10.1001/jamaneurol.2019.4611