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面对进行性多发性硬化症治疗的紧迫性 - 多发性硬化症联盟的建议。

Facing the urgency of therapies for progressive MS - a Progressive MS Alliance proposal.

机构信息

EMD Serono, Billerica, MA, USA.

Department of Computer Science, O'Reilly Institute, Trinity College, Dublin, Ireland.

出版信息

Nat Rev Neurol. 2021 Mar;17(3):185-192. doi: 10.1038/s41582-020-00446-9. Epub 2021 Jan 22.

DOI:10.1038/s41582-020-00446-9
PMID:33483719
Abstract

Therapies for infiltrative inflammation in multiple sclerosis (MS) have advanced greatly, but neurodegeneration and compartmentalized inflammation remain virtually untargeted as in other diseases of the nervous system. Consequently, many therapies are available for the relapsing-remitting form of MS, but the progressive forms remain essentially untreated. The objective of the International Progressive MS Alliance is to expedite the development of effective therapies for progressive MS through new initiatives that foster innovative thinking and concrete advancements. Based on these principles, the Alliance is developing a new funding programme that will focus on experimental medicine trials. Here, we discuss the reasons behind the focus on experimental medicine trials, the strengths and weaknesses of these approaches and of the programme, and why we hope to advance therapies while improving the understanding of progression in MS. We are soliciting public and academic feedback, which will help shape the programme and future strategies of the Alliance.

摘要

多发性硬化症(MS)浸润性炎症的治疗方法已经有了很大的进展,但神经退行性变和分隔性炎症仍然像其他神经系统疾病一样几乎没有针对性。因此,有许多疗法可用于缓解-复发型 MS,但进展型 MS 基本上仍未得到治疗。国际进行性 MS 联盟的目标是通过促进创新思维和具体进展的新举措,加速开发针对进行性 MS 的有效疗法。基于这些原则,该联盟正在开发一项新的资助计划,该计划将侧重于实验医学试验。在这里,我们讨论了关注实验医学试验的原因,这些方法以及该计划的优缺点,以及为什么我们希望在推进治疗方法的同时提高对 MS 进展的认识。我们正在征求公众和学术界的反馈意见,这将有助于制定该计划和联盟的未来战略。

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本文引用的文献

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J Neurol. 2021 Apr;268(4):1474-1484. doi: 10.1007/s00415-020-10315-6. Epub 2020 Nov 20.
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Idebenone does not inhibit disability progression in primary progressive MS.艾地苯醌不会抑制原发性进行性多发性硬化症患者的残疾进展。
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An interspecies translation model implicates integrin signaling in infliximab-resistant inflammatory bowel disease.
用于神经退行性疾病治疗学的杂化纳米结构:生物膜和非生物膜纳米结构结合的艺术。
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A metformin add-on clinical study in multiple sclerosis to evaluate brain remyelination and neurodegeneration (MACSiMiSE-BRAIN): study protocol for a multi-center randomized placebo controlled clinical trial.二甲双胍治疗多发性硬化症的附加临床试验,以评估脑髓鞘再生和神经退行性变(MACSiMiSE-BRAIN):一项多中心随机安慰剂对照临床试验的研究方案。
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