文摘
摘要目的:综合分析平台的理解,弥补和消除肌萎缩性脊髓侧索硬化症(ALS)捕获将集中资源,人才在加拿大推进我们对ALS的生物学基础的理解和解决其异质性。
背景:已经阻碍了肌萎缩性侧索硬化症药物发现的复杂性疾病,临床异质性和罕见的疾病状态。关键的进步将会加速通过多点、多学科协作和共享数据和材料。捕捉ALS将利用网络和扩大现有的临床和基础设施开发的加拿大ALS神经影像财团(CALSNIC)提供最全面的照片,一个人患有肌萎缩性侧索硬化症。这将是推动学术最初通过四个现有的ALS中心在埃德蒙顿,多伦多,蒙特利尔,魁北克城,随后扩展到其他诊所的愿景。
设计/方法:捕捉ALS将提供基础设施和工具,以便收集、存储、分析和传播的标准化,纵向,多维数据和biosamples ALS患者相关疾病和健康对照组。参与者将前瞻性收集详细的临床,神经认知,演讲,和成像数据,伴随着血清,血浆,外周血单核细胞,脑脊液,尿液和尸检材料。将会有一个强烈关注病人接触,获得病人的合作伙伴咨询委员会的建议(PPAC)通过设计和执行过程。
结果:富人通过这个平台采集数据和样本将用于开发和验证新的ALS生物标记和启用多维分层的ALS患者根据临床表型,和成像、基因和细胞特征。操作通过开放科学原则,数据和样品将提供给调查人员和行业合作伙伴。
结论:捕捉ALS将有助于全球努力加快ALS的研究,并提供假设和数据驱动的见解疾病机制和临床异质性。
披露:卡尔拉博士的机构已经收到了来自大脑研究支持加拿大的基础。塔尼亚Bubela已收到个人薪酬在5000 - 9999美元的范围作为一个军官或董事会成员研究所卫生系统转换和可持续性。塔尼亚Bubela的机构收到加拿大基因组的研究支持。塔尼亚Bubela的机构已经收到了来自大脑研究支持加拿大。塔尼亚Bubela收到了研究的机构支持创新、科学和经济的发展,加拿大。迪普雷博士已经收到个人薪酬在500 - 4999美元的范围为在科学咨询服务或数据安全监测委员会Akcea疗法加拿大。迪普雷博士的机构已经接到ARSACS基础研究支持。迪普雷博士的机构已经接到CHUdeQuebec基础研究支持。埃文斯博士已经收到biospective的个人作为一个雇员补偿。埃文斯博士已经收到个人薪酬在10000 - 49999美元的范围为biospective担任顾问。 Dr. Genge has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for AL-S Pharma. Dr. Genge has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. Dr. Genge has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Cytokinetics. Dr. Genge has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amylyx. Dr. Genge has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Quralis. Dr. Genge has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Genge has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for MTPA. Dr. Genge has received personal compensation in the range of $500-$4,999 for serving as a Consultant for WAVE. Russell Greiner has nothing to disclose. Dr. Johnston has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Mitsubishi Tanabe. Dr. Johnston has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen Inc.. Dr. Johnston has a non-compensated relationship as a Board Member with ALS Canada that is relevant to AAN interests or activities. Dr. Johnston has a non-compensated relationship as a Chair with Canadian ALS Clinical Research Group that is relevant to AAN interests or activities. Janice Robertson has nothing to disclose. Ekaterina Rogaeva has nothing to disclose. David C. Taylor, MD has nothing to disclose. Christine Vande Velde has received personal compensation in the range of $500-$4,999 for serving as a Consultant for BridgeBio. Christine Vande Velde has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for MT Pharma. Christine Vande Velde has received research support from ALS Canada. Christine Vande Velde has received research support from Target ALS . Christine Vande Velde has received research support from Muscular Dystrophy Association. Christine Vande Velde has received research support from ALS Association. Dr. Zinman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen and MTP. Dr. Zinman has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen and MTP. Hannah Kaneb has nothing to disclose.
