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多发性硬化症新型和新兴疾病修正疗法及干细胞疗法的疗效与安全性结果更新:综述

Updates on efficacy and safety outcomes of new and emerging disease modifying therapies and stem cell therapy for Multiple Sclerosis: A review.

作者信息

Peterson Sarah, Jalil Amaris, Beard Katherine, Kakara Mihir, Sriwastava Shitiz

机构信息

West Virginia University, School of Medicine, Morgantown, WV, USA; Department of Neurology, Rockefeller Neuroscience Institute, West Virginia University, Morgantown, WV, USA; Department of Neurology, University of Cincinnati Medical Center, Cincinnati, OH, USA.

West Virginia University, School of Medicine, Morgantown, WV, USA; Department of Neurology, Rockefeller Neuroscience Institute, West Virginia University, Morgantown, WV, USA.

出版信息

Mult Scler Relat Disord. 2022 Dec;68:104125. doi: 10.1016/j.msard.2022.104125. Epub 2022 Aug 31.

Abstract

Multiple Sclerosis (MS) is a chronic neurodegenerative autoimmune disorder of the central nervous system (CNS) and the most common cause of serious physical disability in working-age adults. Drug development and research in this field have rapidly evolved over the past two decades, leading to the broad array of treatment options available today. These disease-modifying therapies (DMTs) work through distinct mechanisms of action and exhibit varying safety and efficacy profiles to help manage symptoms and reduce exacerbations in MS patients. Our extensive understanding of this condition has also led to novel approaches, such as the discovery of specific biomarkers that allow us to monitor the therapeutic response towards DMTs. The development of new DMTs continues to progress quickly today, and it can be difficult for clinicians to remain up to date on the most recent advancements and new treatment options for their patients. In this comprehensive review, we provide an outline of current MS medications in the pipeline including emerging DMTs and stem cell therapy, as well as the unique characteristics of these medications, including their indications, pharmacokinetic effects, and the relevant advancements.

摘要

多发性硬化症(MS)是一种中枢神经系统(CNS)的慢性神经退行性自身免疫性疾病,是工作年龄成年人严重身体残疾的最常见原因。在过去二十年中,该领域的药物开发和研究迅速发展,产生了如今可用的广泛治疗选择。这些疾病修正疗法(DMTs)通过不同的作用机制发挥作用,并表现出不同的安全性和疗效特征,以帮助管理MS患者的症状并减少病情加重。我们对这种疾病的广泛了解还催生了新的方法,例如发现特定的生物标志物,使我们能够监测对DMTs的治疗反应。如今,新DMTs的开发仍在迅速推进,临床医生可能难以跟上其患者最新进展和新治疗选择的步伐。在这篇全面综述中,我们概述了正在研发的当前MS药物,包括新兴的DMTs和干细胞疗法,以及这些药物的独特特征,包括其适应症、药代动力学效应和相关进展。

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