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帕金森病治疗策略的研究进展。

Progress towards therapies for disease modification in Parkinson's disease.

机构信息

Department of Clinical and Movement Neurosciences, UCL Queen Square Institute of Neurology, London, UK; The National Hospital for Neurology and Neurosurgery, Queen Square, London, UK.

Parkinson's Disease and Movement Disorders Center, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.

出版信息

Lancet Neurol. 2021 Jul;20(7):559-572. doi: 10.1016/S1474-4422(21)00061-2.

Abstract

The development of interventions to slow or halt the progression of Parkinson's disease remains a priority for patients and researchers alike. To date, no agents have been shown to have unequivocal evidence of disease-modifying effects in Parkinson's disease. The absence of disease-modifying treatments might relate not only to inadequate approaches for the selection of therapeutic candidates but also to insufficient attention to detail in clinical trial design. Better understanding of Parkinson's disease pathogenesis associated with advances in laboratory models, the use of objective biomarkers of disease progression and target engagement, and a focus on agents known to be safe for human use, alongside the use of precision medicine approaches, should together greatly increase the likelihood for successful identification of disease-modifying treatments for Parkinson's disease.

摘要

开发干预措施以减缓或阻止帕金森病进展仍然是患者和研究人员的首要任务。迄今为止,尚无药物被证明对帕金森病具有明确的疾病修饰作用。缺乏疾病修饰治疗的原因不仅可能与治疗候选药物选择的方法不当有关,还可能与临床试验设计中对细节的关注不够有关。随着实验室模型相关帕金森病发病机制的进展、疾病进展和目标结合的客观生物标志物的使用,以及对已知对人体安全的药物的关注,以及使用精准医疗方法,应共同极大地增加识别帕金森病疾病修饰治疗的可能性。

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