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.

Dr. Jeste is a behavioral child neurologist specializing in autism and related neurodevelopmental disorders. She is an Associate Professor-in-Residence in Psychiatry, Neurology and Pediatrics at the UCLA David Geffen School of Medicine, the director of the UCLA CARING Clinic, and a lead investigator in the UCLA Center for Autism Research and Treatment (CART). Dr. Jeste’s research is focused on developing methods to improve precision in the diagnosis and treatment of neurodevelopmental disorders. Her lab studies neurodevelopmental disorders from early infancy through late childhood. She holds several national and international leadership positions including the Board of Directors of the American Brain Foundation, Board of Directors of the National Organization for Rare Disorders, and the Board of Directors of the International Society for Autism Research. In 2019 she became Chair of the International Baby Siblings Research Consortium. In 2019 she was awarded the Presidential Early Career Award for Scientists and Engineers for her innovations in research in early predictors and intervention for genetic neurodevelopmental disorders.