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International collaboration including patients is essential to develop new therapies for patients with myositis

机译:包括患者在内的国际合作对于为肌炎患者开发新的疗法至关重要

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Purpose of reviewTo discuss the needs for international collaborations between Investigators in different disciplines working with myositis and with patients with myositis.Recent findingsRecent advances in detection of several myositis-specific autoantibodies that are associated with distinct clinical phenotypes, will enable studies in new well defined clinically homogenous subgroups of myositis This is likely to lead to development of new information on molecular pathogenesis that might be different in different myositis subgroups. Subgrouping patients according to autoantibody profile may also be important to assess outcome, to identify prognostic biomarkers and in clinical trials. As these are rare disorders international collaboration is essential to enrol large enough cohorts of the subgroups. To facilitate such collaboration we have developed a web-based international myositis register, www.euromyositis.eu, which includes validated outcome measures and patient reported outcome measures. This register is to support research but also to support decision-making in the clinic. We welcome investigators to join the Euromyositis register.SummaryMyositis is a heterogeneous disorder with varying treatment response and outcome. There is a high unmet need for new therapies which can only be achieved by increased knowledge on molecular disease mechanisms. Subgrouping patients according to autoantibody profile may be a new way forward to get a better understanding on disease mechanisms and to develop novel therapies.
机译:审查的目的讨论了使用肌炎和肌炎患者的不同学科中调查人员之间的国际合作的需求。特定发现患者检测与不同临床表型相关的几种肌炎特异性自身抗体的进步,将在临床上进行研究。 Myositis的均质亚组,这可能导致关于分子发病机制的新信息,其在不同的肌炎亚组中可能不同。根据自身抗体谱的亚组患者也可能对评估结果来评估,以鉴定预后生物标志物和临床试验。由于这些是罕见的疾病,国际合作对于注册足够的亚组队列至关重要。为了促进这种协作,我们开发了一个基于网络的国际肌炎,www.euromyositis.eu,包括验证的结果措施和患者报告的结果措施。该登记册是支持研究,还支持诊所的决策。我们欢迎调查人员加入Euroomyositis Register.SummaryMyositis是一种异质疾病,治疗响应和结果不同。对于新的疗法,有一个高的未满足需要通过增加对分子疾病机制的知识来实现​​的。根据自身抗体剖面的亚组患者可能是一种新的方式,以更好地了解疾病机制并开发新疗法。

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