Biologically defined neuronal synuclein disease as a tool to advance drug development

IF 8.2 1区 医学 Q1 NEUROSCIENCES NPJ Parkinson's Disease 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
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Abstract

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.
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将生物定义的神经元突触核蛋白疾病作为推动药物开发的工具
在最近的一篇观点文章(JAMA Neurol. 2024;81:789-90)中,Okubadejo 等人对最近提出的两种突触核蛋白病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,我们提出了另一种观点--此类框架是向前迈出的重要一步,它允许针对生物定义的人群进行精确治疗,而精确治疗可以使用特定阶段的结果进行准确评估。
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来源期刊
NPJ Parkinson's Disease
NPJ Parkinson's Disease Medicine-Neurology (clinical)
CiteScore
9.80
自引率
5.70%
发文量
156
审稿时长
11 weeks
期刊介绍: npj Parkinson's Disease is a comprehensive open access journal that covers a wide range of research areas related to Parkinson's disease. It publishes original studies in basic science, translational research, and clinical investigations. The journal is dedicated to advancing our understanding of Parkinson's disease by exploring various aspects such as anatomy, etiology, genetics, cellular and molecular physiology, neurophysiology, epidemiology, and therapeutic development. By providing free and immediate access to the scientific and Parkinson's disease community, npj Parkinson's Disease promotes collaboration and knowledge sharing among researchers and healthcare professionals.
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