生物学定义的神经元α-突触核蛋白病作为推进药物研发的工具
Biologically defined neuronal synuclein disease as a tool to advance drug development.
作者信息
Pagano Gennaro, Dam Tien, Kerchner Geoffrey A, Galpern Wendy R, Biagioni Milton, Karan Rajesh, Jennings Danna, Peterschmitt M Judith, Nikolcheva Tania, Brundin Patrik
机构信息
Roche Pharma Research and Early Development (pRED), Roche Innovation Center, F. Hoffmann-La Roche Ltd., Basel, Switzerland.
University of Exeter Medical School, London, UK.
出版信息
NPJ Parkinsons Dis. 2024 Dec 20;10(1):235. doi: 10.1038/s41531-024-00845-5.
In a recent Viewpoint article (. 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.
在最近的一篇观点文章（《神经病学年鉴》，2024年；81卷：789 - 90页）中，奥库巴德乔等人对最近提出的两种关于突触核蛋白病的生物学定义和分期系统（神经元突触核蛋白病综合分期系统和SynNeurGe系统）表示担忧。在承认这些担忧的同时，我们提供另一种观点——即这样的框架代表了重要的进步，因为它们允许针对生物学定义的人群进行精准治疗，并能够使用特定阶段的结果进行准确评估。
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