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Path to Parkinson Disease Prevention: Conclusion and Outlook.
Neurology ( IF 9.9 ) Pub Date : 2022-08-16 , DOI: 10.1212/wnl.0000000000200793
Daniela Berg 1 , Grace F Crotty 1 , Jessi L Keavney 1 , Michael A Schwarzschild 1 , Tanya Simuni 1 , Caroline Tanner 1
Affiliation  

Tremendous progress in our understanding of the pathophysiology and clinical manifestations of the prodromal phase of Parkinson disease (PD) offers a unique opportunity to start therapeutic interventions as early as possible to slow or even stop the progression to clinically manifest motor PD. A Parkinson's Prevention Conference, "Planning for Prevention of Parkinson's: A trial design symposium and workshop" was convened to discuss all issues that need to be addressed before the launch of the first PD prevention study. In this review, we summarize the major opportunities and challenges in designing prevention trials in PD, organized by the following critical trial design questions: Who (should be enrolled)? What (to test)? How (to measure prevention)? and the pivotal question, When during the prodromal disease (should we start these trials)? We outline the implications of these questions and their meaning for a responsible, sustainable, and fruitful further planning for prevention trials. Despite the great progress that has been made, it needs to be acknowledged that several queries remain to be carefully considered and addressed because prevention trials are being planned and become a reality.

中文翻译:

帕金森病预防之路:结论和展望。

我们对帕金森病 (PD) 前驱期的病理生理学和临床表现的理解取得了巨大进展,这为尽早开始治疗干预以减缓甚至阻止临床表现的运动 PD 进展提供了独特的机会。召开了一次帕金森氏症预防会议,“帕金森氏症预防计划:试验设计研讨会和研讨会”,以讨论在启动第一个 PD 预防研究之前需要解决的所有问题。在这篇综述中,我们总结了设计 PD 预防试验的主要机遇和挑战,由以下关键试验设计问题组织:谁(应该参加)?什么(测试)?如何(衡量预防)?和关键的问题,在前驱疾病期间(我们应该开始这些试验)?我们概述了这些问题的含义及其对负责任、可持续和富有成效的预防试验进一步规划的意义。尽管已经取得了很大进展,但需要承认的是,仍有几个问题需要仔细考虑和解决,因为预防试验正在计划中并成为现实。
更新日期:2022-08-16
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