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SCIENTIFIC LITERATURE · MED

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

J Neural Transm (Vienna) · 2022-05-31

DOI: 10.1007/s00702-022-02511-7PMID: 35639160

Open accessResearch Support, Non-U.S. Gov't; review-article; Review; Journal ArticleOriginal source ↗

Research overview

The text below is the source article’s original abstract under a verified reuse license. cc by

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.

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