Ambroxol as a disease-modifying treatment to reduce the risk of cognitive impairment in GBA-associated Parkinson’s disease: a multicentre, randomised, double-blind, placebo-controlled, phase II trial. The AMBITIOUS study protocol

BACKGROUND: Heterozygous mutations in the GBA gene, encoding the lysosomal enzyme β-glucocerebrosidase (GCase), are the most frequent genetic risk factor for Parkinson’s disease (PD). GBA-related PD (GBA-PD) patients have higher risk of dementia and reduced survival than non-carriers. Preclinical st...

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Detalles Bibliográficos
Autores principales: Colucci, Fabiana, Avenali, Micol, De Micco, Rosita, Fusar Poli, Marco, Cerri, Silvia, Stanziano, Mario, Bacila, Ana, Cuconato, Giada, Franco, Valentina, Franciotta, Diego, Ghezzi, Cristina, Gastaldi, Matteo, Elia, Antonio Emanuele, Romito, Luigi, Devigili, Grazia, Leta, Valentina, Garavaglia, Barbara, Golfrè Andreasi, Nico, Cazzaniga, Federico, Reale, Chiara, Galandra, Caterina, Germani, Giancarlo, Mitrotti, Pierfrancesco, Ongari, Gerardo, Palmieri, Ilaria, Picascia, Marta, Pichiecchio, Anna, Verri, Mattia, Esposito, Fabrizio, Cirillo, Mario, Di Nardo, Federica, Aloisio, Simone, Siciliano, Mattia, Prioni, Sara, Amami, Paolo, Piacentini, Sylvie, Bruzzone, Maria Grazia, Grisoli, Marina, Moda, Fabio, Eleopra, Roberto, Tessitore, Alessandro, Valente, Enza Maria, Cilia, Roberto
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2023
Materias:
Protocol
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10679992/
https://www.ncbi.nlm.nih.gov/pubmed/38027469
http://dx.doi.org/10.1136/bmjno-2023-000535

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