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

• 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
• 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

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.

项目摘要/摘要 共济失调是一种致残性和致命性神经系统疾病，由多种遗传和后天原因引起。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模式”将汇集一个国际研究人员名册，以解决共济失调的各种原因，更好地定义共济失调的致病基础，探索治疗路线，检查生物标记物的发现和实施问题，促进共济失调的强有力的试验，并帮助在共济失调研究领域建立未来的领导者。会议将集中讨论最新的科学进展和新出现的综合治疗方法，目标如下：1)加深我们对小脑功能和功能障碍的理解；2)确定神经保护目标和策略；3)促进在共济失调方面进行强有力的临床试验；4)通过促进年轻研究人员的参与，帮助建立未来的共济失调研究领导者；以及5)使受训人员与共济失调患者及其家人接触。AIM 2020通过创建一个论坛来促进共济失调研究人员和临床医生之间的合作，并通过支持关于共济失调研究和治疗方法的讨论来满足共济失调社区的关键需求，这在该领域正在走向多中心临床试验的情况下尤其重要。AIM 2020会议是国内最大的共济失调患者基金会年会的附属和重叠会议，在同一酒店举行，通过让共济失调研究人员与患者和家属直接接触，将最大限度地发挥这次会议对科学家和患者的影响。目的2020年将集中在共济失调研究和治疗方法方面的最新进展，特别是与正在进行的临床试验组织有关的生物标记物的发现和实施。共济失调被广泛定义为失去协调的运动控制，可影响人类运动的方方面面：步态、灵巧度、言语、吞咽和眼球运动，全世界每2000人中就有1人患有共济失调。一种新的对共济失调的遗传学理解已经确定了这些疾病中重叠的致病机制，促进了共同的治疗方法。疾病目标和治疗策略中的这些新概念要求科学家、临床医生和患者之间加强沟通和合作，以便能够成功地开发治疗方法，这将是AIM 2020的主要目标。AIM 2020会议还将提供一个论坛，支持研究生和其他受训人员的职业发展，同时招募这一领域的新研究人员，这对于将转化研究进展推进到有意义的疗法至关重要。