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用于治疗帕金森病的新型靶向疗法。

Novel targeted therapies for Parkinson's disease.

机构信息

Department of Clinical Neuroscience, Karolinska Institutet, Stockholm, Sweden.

Center of Neurology, Academic Specialist Center, Solnavägen 1E, 113 65, Stockholm, Sweden.

出版信息

Mol Med. 2021 Feb 25;27(1):17. doi: 10.1186/s10020-021-00279-2.

Abstract

Parkinson's disease (PD) is the second more common neurodegenerative disease with increasing incidence worldwide associated to the population ageing. Despite increasing awareness and significant research advancements, treatment options comprise dopamine repleting, symptomatic therapies that have significantly increased quality of life and life expectancy, but no therapies that halt or reverse disease progression, which remain a great, unmet goal in PD research. Large biomarker development programs are undertaken to identify disease signatures that will improve patient selection and outcome measures in clinical trials. In this review, we summarize PD-related mechanisms that can serve as targets of therapeutic interventions aiming to slow or modify disease progression, as well as previous and ongoing clinical trials in each field, and discuss future perspectives.

摘要

帕金森病(PD)是全球第二常见的神经退行性疾病,发病率随着人口老龄化而增加。尽管人们的认识不断提高,研究也取得了重大进展,但治疗方法仍仅限于补充多巴胺的对症治疗,这些治疗方法显著提高了生活质量和预期寿命,但没有任何能够阻止或逆转疾病进展的疗法,这仍然是 PD 研究中的一个重大未满足的目标。目前正在开展大规模的生物标志物开发计划,以确定疾病特征,从而改善临床试验中的患者选择和结果衡量标准。在这篇综述中,我们总结了 PD 相关的机制,这些机制可以作为治疗干预的靶点,旨在减缓或改变疾病进展,以及每个领域的既往和正在进行的临床试验,并讨论了未来的前景。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9ed5/7905684/60f370ce4529/10020_2021_279_Fig1_HTML.jpg

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