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Biologically defined neuronal synuclein disease as a tool to advance drug development
npj Parkinson's Disease ( IF 6.7 ) Pub Date : 2024-12-20 , DOI: 10.1038/s41531-024-00845-5
Gennaro Pagano, Tien Dam, Geoffrey A. Kerchner, Wendy R. Galpern, Milton Biagioni, Rajesh Karan, Danna Jennings, M. Judith Peterschmitt, Tania Nikolcheva, Patrik Brundin

In a recent Viewpoint article (JAMA Neurol. 2024;81:789‒90), Okubadejo et al. raised concerns regarding two recent proposals for biological definitions and staging systems for synucleinopathies (the Neuronal Synuclein Disease Integrated Staging System and SynNeurGe system). While acknowledging these concerns, we provide an alternative perspective—that such frameworks represent important steps forward by allowing biologically defined populations to be targeted with precision treatments that can be accurately evaluated using stage-specific outcomes.

中文翻译:


生物学定义的神经元突触核蛋白病是推进药物开发的工具



在最近的一篇 Viewpoint 文章(JAMA Neurol. 2024;81:789\u201290),Okubadejo 等人对最近提出的两个突触核蛋白病的生物学定义和分期系统(神经元突触核蛋白疾病综合分期系统和 SynNeurGe 系统)提出了担忧。在承认这些担忧的同时,我们提供了另一种观点——这样的框架代表了向前迈出的重要一步,它允许以精确治疗为目标,这些治疗可以使用特定阶段的结果进行准确评估。
更新日期:2024-12-20
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