Codrin Lungu et al.
In October 2018, the National Institute of Neurological Disorders and Stroke (NINDS) conducted a workshop entitled “Defining Emergent Opportunities in Dystonia Research,” cosponsored by the Dystonia Medical Research Foundation, and including representatives from academic institutions, industry, patient advocacy groups, and funding and regulatory agencies.
The overall goals were to (1) outline the current understanding of the natural history, pathology, and therapeutic space in dystonia; (2) identify gaps and limitations in treatment; and (3) generate research priorities for dystonia. Sessions included the natural history of the disease, defining the underlying etiology, elucidating the pathophysiology, developing research technologies, building and evaluating research resources, refining therapeutic approaches, and finally prioritizing dystonia research recommendations.
The group identified as a top research priority the leveraging of existing resources. These include the NINDS Human Genetics Resource Center. To date, the Dystonia Sub-Collection of the NINDS Human Genetics Resource Center has received over 3,000 submissions which are currently available for general research use.
All outlined priorities for dystonia research are only achievable through close and continuing collaborations among all stakeholders, including across research teams. Isolated approaches are unlikely to yield the advances in knowledge and therapeutic approaches that the patient with dystonia and the advocacy community expect. Participants expressed enthusiasm for collaborative work.
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