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

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.

Automated summary

This review provides an overview of GBA-associated Parkinson's disease, focusing on clinical trajectories, pathological mechanisms, and treatment options. The severity of mutations influences disease characteristics, and these findings will guide patient stratification and clinical trial design in the future.