“Clinical trial readiness for neurodevelopmental disorders: On the road to precision health”
Hosted by the Department of Psychiatry and the Intellectual and Developmental Disability Research Center (IDDRC).
Abstract: We have entered an era of precision therapeutics in neurodevelopmental disorders (NDD), with precision both in timing of our diagnoses and in the identification of specific genetic etiologies that cause NDDs. As disease-specific therapies are developed, we must be prepared for the design and implementation of successful clinical trials. This clinical trial readiness includes (1) identification of meaningful clinical endpoints, (2) measurement of biomarkers that inform patient selection and drug target engagement measurement, and (3) methods to improve scalability and accessibility of research and treatment for families. This presentation will begin with an overview of the state of the field in precision health in NDDs followed by data from several studies that have addressed the goals listed above in clinical trial readiness, including prospective studies of high risk infants and endpoint and biomarker studies in syndromic NDDs.
This lecture is part of the Developmental Psychopathology Series, which is integrated as part of the Department of Psychiatry Research Seminar.
For inquiries contact Daniel Gray.