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

Colucci, F; Avenali, M; De Micco, R; Poli, MF; Cerri, S; Stanziano, M; Bacila, A; Cuconato, G; Franco, V; Franciotta, D; Ghezzi, C; Gastaldi, M; Elia, AE; Romito, L; Devigili, G; Leta, V; Garavaglia, B; Andreasi, NG; Cazzaniga, F; Reale, C; Galandra, C; Germani, G; Mitrotti, P; Ongari, G; Palmieri, I; Picascia, M; Pichiecchio, A; Verri, M; Esposito, F; Cirillo, M; Di Nardo, F; Aloisio, S; Siciliano, M; Prioni, S; Amami, P; Piacentini, S; Bruzzone, MG; Grisoli, M; Moda, F; Eleopra, R; Tessitore, A; Valente, EM; Cilia, R

Cilia, R (通讯作者),Fdn IRCCS Ist Neurol Carlo Besta, Dept Clin Neurosci, Parkinson & Movement Disorders Unit, Milan, Italy.

BMJ NEUROLOGY OPEN, 2023; 5 (2):

Abstract

Background Heterozygous mutations in the GBA gene, encoding the lysosomal enzyme beta-glucocerebrosidase (GCase), are the most frequent genetic risk f......

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