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未来已来:澳大利亚视角下帕金森病及前驱疾病的疾病修饰治疗试验

Future is now: an Australasian perspective on disease-modifying trials in Parkinson's and prodromal disease.

作者信息

Lewis Simon Jg, Sue Carolyn M, Cooper Antony, Halliday Glenda M

机构信息

Macquarie Medical School, Macquarie University, Sydney, New South Wales, Australia.

Kinghorn Chair, Neurodegeneration, Neuroscience Research Australia, Randwick, New South Wales, Australia.

出版信息

BMJ Neurol Open. 2025 Mar 21;7(1):e001070. doi: 10.1136/bmjno-2025-001070. eCollection 2025.

Abstract

There is increasing interest in the role of platform trials, where several investigational products targeting disease modification in Parkinson's Disease can be assessed in parallel. Indeed, several initiatives are currently gearing up across North America and Europe to conduct such studies. However, to date, little attention has been paid to ongoing efforts that already exist in Australia and look soon to expand across to New Zealand as part of greater collaboration. This viewpoint will highlight some of these ongoing efforts addressing the challenges and potential solutions for delivering successful studies.

摘要

人们对平台试验的作用越来越感兴趣,通过平台试验可以同时评估几种针对帕金森病疾病修饰的研究性产品。事实上,目前北美和欧洲的几项举措正在加快推进以开展此类研究。然而,迄今为止,澳大利亚已经开展的相关工作却很少受到关注,并且这些工作很快将作为更大规模合作的一部分扩展到新西兰。本文观点将重点介绍其中一些正在进行的工作,探讨开展成功研究面临的挑战及潜在解决方案。

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