Ataxia Investigators Meeting 8: Leveraging Therapeutic Opportunity into Novel Treatment Paradigms

共济失调研究者会议 8：利用治疗机会开发新型治疗范式

基本信息

批准号：
9913421
负责人：
ALBERT R LA SPADA
金额：
$ 2万
依托单位：
DUKE UNIVERSITY
依托单位国家：
美国
项目类别：
财政年份：
2020
资助国家：
美国
起止时间：
2020-03-01 至 2021-02-28
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/9913421
关键词：
Address Affect Ataxia Basic Science Biological Products Categories Clinical Trials Collaborations Communication Communities Country Deglutition Development Disease Disease Pathway Disease model Drug Industry Economics Ensure Etiology Eye Movements Family Foundations Functional disorder Funding Future Gait Genetic Goals Human Individual International Location Molecular Movement Multi-Institutional Clinical Trial Nature Neurodegenerative Disorders Pathogenicity Patients Prevalence Process Research Research Personnel Route Scientific Advances and Accomplishments Scientist Shark Speech Testing Therapeutic Time Translating Translational Research Travel Underrepresented Minority United States National Institutes of Health Validation Woman Work biomarker discovery career development clinical care clinical phenotype design dexterity effective therapy graduate student lectures meetings minority investigator motor control multidisciplinary nervous system disorder novel novel therapeutics posters predictive marker prevent programs recruit small molecule stem success symposium targeted treatment therapy development translational approach

项目摘要

PROJECT SUMMARY/ABSTRACT Ataxia, a disabling and frequently fatal neurological disorder, results from a wide variety of genetic and acquired etiologies. The 8th Ataxia Investigators’ Meeting, “AIM 2020: Leveraging Therapeutic Opportunity into Novel Treatment Paradigms” will assemble an international roster of investigators to address the diverse causes of ataxia, better define the pathogenic basis of ataxia, explore routes to therapy, examine issues of biomarker discovery and implementation, facilitate robust trials in ataxias, and help to establish future leaders in the ataxia research field. The conference will focus on the most recent scientific advances and emerging integrative approaches toward therapy, with the following objectives: 1) refine our understanding of cerebellar function and dysfunction; 2) define neuroprotective targets and strategies; 3) facilitate development of robust clinical trials in ataxias; 4) help to establish future leaders of ataxia research by facilitating the involvement of young investigators; and 5) bring trainees into contact with ataxia patients and their families. AIM 2020 fulfills a crucial need for the ataxia community by creating a forum to facilitate collaboration among ataxia researchers and clinicians, and by enabling discussion on ataxia research and therapeutic approaches, which is especially important now that the field is moving towards multi-center clinical trials. The AIM 2020 meeting, affiliated and overlapping with the annual meeting of the largest ataxia patient foundation in the country occurring at the same hotel, will maximize the impact of this meeting for scientists and patients alike by bringing ataxia researchers into direct contact with patients and families. AIM 2020 will focus on the most recent advances in ataxia research and therapeutic approaches for ataxia disorders, with a particular new emphasis on biomarker discovery and implementation in association with ongoing clinical trial organization. Ataxia, which is broadly defined as the loss of coordinated motor control, can affect all aspects of human movement: gait, dexterity, speech, swallowing, and eye movements, and afflicts ~1 in 2,000 individuals worldwide. An emerging genetic understanding of ataxias has identified overlapping pathogenic mechanisms in these disorders, facilitating shared therapeutic approaches. These new concepts in disease targets and therapeutic strategies demand increased communication and collaboration among scientists, clinicians, and patients, so that therapies can be successfully developed, and this will be a primary goal of AIM 2020. The AIM 2020 meeting will also provide a forum for supporting the career development of graduate student and fellow trainees, while recruiting new investigators into this field, which is critical for advancing translational research advances into meaningful therapies.

项目概要/摘要共济失调是一种致残且常常致命的神经系统疾病，由多种遗传和后天病因引起。第八届共济失调研究人员会议“AIM 2020：利用治疗机会进入新的治疗范式”将汇集一个国际研究人员名册来解决这一问题。共济失调的多种原因，更好地确定共济失调的致病基础，探索治疗途径，检查生物标志物发现和实施的问题，促进稳健的试验会议将重点关注最新的科学进展和新兴的综合治疗方法，其目标如下：1）完善我们对小脑功能和功能障碍的理解；2）定义共济失调。神经保护目标和策略；3）促进共济失调临床试验的发展；4）通过促进年轻研究人员的参与，帮助建立共济失调研究的未来领导者；5）让受训者接触AIM 2020 通过创建一个论坛来促进共济失调研究人员和信徒之间的合作，并通过讨论共济失调研究和治疗方法来满足共济失调社区的重要需求，这一点在该领域正在不断发展的情况下尤为重要。 AIM 2020 会议与国内最大的共济失调患者基金会年会在同一家酒店举行，并重叠举行，将最大限度地发挥本次会议对科学家和患者的影响。同样，AIM 2020 将重点关注共济失调研究和共济失调疾病治疗方法的最新进展，特别是与正在进行的临床试验组织相关的生物标志物发现和实施。共济失调被广义地定义为协调运动控制的丧失，可以影响人类运动的各个方面：步态、灵巧性、言语、吞咽和眼球运动，大约每 2,000 人中就有 1 人患有共济失调对共济失调的新兴遗传学认识已经确定了这些疾病的重叠致病机制，促进了疾病目标和治疗策略的共同治疗方法，需要科学家、主教和患者之间加强沟通和合作，以便能够进行治疗。成功开发，这将是 AIM 2020 的首要目标。AIM 2020 会议还将提供一个论坛，支持研究生和学员的职业发展，同时招募新的研究人员进入该领域，这对于推进转化研究至关重要进步转化为有意义的疗法。