Unlocking the mystery of biomarkers: A brief introduction, challenges and opportunities in Parkinson Disease

揭开生物标志物的奥秘:帕金森病研究的简要介绍、挑战与机遇

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Abstract

First described 200 years ago, Parkinson Disease (PD) exhibits considerable heterogeneity in clinical presentation, as well as trajectory of motor and non-motor decline. This heterogeneity, in turn, complicates the planning of clinical research, particularly trials of disease-modifying therapies, as well as the care of PD patients. While clinical features have been used to delineate subgroups of PD patients, clinical subtyping is hampered by change in features over time, and clinical subtyping may fail to capture the biological processes underlying heterogeneity. In contrast, biomarkers - objective measures that serve as indicators of normal biological processes, pathogenic processes, or pharmacologic responses to therapeutic interventions - have promise to delineate molecularly-defined subgroups of PD patients who may be most likely to benefit from specific therapeutic interventions. Here we review the present role of genetic and biochemical biomarkers in PD. Moreover, we highlight areas where the use of biomarkers may benefit clinical trial planning, as well as clinical care through the application of a "precision medicine" approach, in the near term.

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