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

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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.