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GBA-associated PD: chances and obstacles for targeted treatment strategies.

ABSTRACT: Given the clear role of GBA in the pathogenesis of Parkinson's disease (PD) and its impact on phenotypical characteristics, this review provides an overview of the current knowledge of GBA-associated PD with a special focus on clinical trajectories and the underlying pathological mechanisms. Importantly, differences and characteristics based on mutation severity are recognized, and current as well as potential future treatment options are discussed. These findings will inform future strategies for patient stratification and cohort enrichment as well as suitable outcome measures when designing clinical trials.

SUBMITTER: Hoglinger G

PROVIDER: S-EPMC9463270 | biostudies-literature | 2022 Sep

REPOSITORIES: biostudies-literature

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GBA-associated PD: chances and obstacles for targeted treatment strategies.

Höglinger Günter G Schulte Claudia C Jost Wolfgang H WH Storch Alexander A Woitalla Dirk D Krüger Rejko R Falkenburger Björn B Brockmann Kathrin K

Journal of neural transmission (Vienna, Austria : 1996) 20220531 9

Given the clear role of GBA in the pathogenesis of Parkinson's disease (PD) and its impact on phenotypical characteristics, this review provides an overview of the current knowledge of GBA-associated PD with a special focus on clinical trajectories and the underlying pathological mechanisms. Importantly, differences and characteristics based on mutation severity are recognized, and current as well as potential future treatment options are discussed. These findings will inform future strategies f ...[more]

PMID: 35639160

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