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神经肌肉疾病的新型对比剂、靶点及磁共振成像和波谱技术的探索——COST 行动 BM1304 MYO-MRI 生物医学和分子生物科学工作组 3 的研讨会报告。

Exploration of New Contrasts, Targets, and MR Imaging and Spectroscopy Techniques for Neuromuscular Disease - A Workshop Report of Working Group 3 of the Biomedicine and Molecular Biosciences COST Action BM1304 MYO-MRI.

机构信息

Amsterdam UMC, University of Amsterdam, Amsterdam, The Netherlands.

NMR Laboratory, Neuromuscular Investigation Center, Institute of Myology & NMR Laboratory, CEA/DRF/IBFJ/MIRCen, Paris, France.

出版信息

J Neuromuscul Dis. 2019;6(1):1-30. doi: 10.3233/JND-180333.

Abstract

Neuromuscular diseases are characterized by progressive muscle degeneration and muscle weakness resulting in functional disabilities. While each of these diseases is individually rare, they are common as a group, and a large majority lacks effective treatment with fully market approved drugs. Magnetic resonance imaging and spectroscopy techniques (MRI and MRS) are showing increasing promise as an outcome measure in clinical trials for these diseases. In 2013, the European Union funded the COST (co-operation in science and technology) action BM1304 called MYO-MRI (www.myo-mri.eu), with the overall aim to advance novel MRI and MRS techniques for both diagnosis and quantitative monitoring of neuromuscular diseases through sharing of expertise and data, joint development of protocols, opportunities for young researchers and creation of an online atlas of muscle MRI and MRS. In this report, the topics that were discussed in the framework of working group 3, which had the objective to: Explore new contrasts, new targets and new imaging techniques for NMD are described. The report is written by the scientists who attended the meetings and presented their data. An overview is given on the different contrasts that MRI can generate and their application, clinical needs and desired readouts, and emerging methods.

摘要

神经肌肉疾病的特征是肌肉进行性退化和肌肉无力,导致功能障碍。虽然这些疾病中的每一种都很少见,但作为一个整体,它们很常见,而且绝大多数都缺乏完全获得市场批准的药物的有效治疗方法。磁共振成像和光谱技术(MRI 和 MRS)在这些疾病的临床试验中作为一种结果测量方法,显示出越来越大的希望。2013 年,欧盟资助了名为 MYO-MRI(www.myo-mri.eu)的 COST(科学技术合作)行动 BM1304,其总体目标是通过共享专业知识和数据、联合制定方案、为年轻研究人员提供机会以及创建肌肉 MRI 和 MRS 的在线图谱,来推进用于神经肌肉疾病诊断和定量监测的新型 MRI 和 MRS 技术。在这份报告中,介绍了工作组 3 讨论的主题,该工作组的目标是:探索用于神经肌肉疾病的新对比剂、新靶点和新成像技术。该报告由参加会议并介绍其数据的科学家撰写。报告概述了 MRI 可以产生的不同对比剂及其应用、临床需求和预期结果,以及新兴方法。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f297/6398566/5abe03bed927/jnd-6-jnd180333-g001.jpg

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