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| Targeted Medication Treatments for Behavioral Features of Autism and Fragile X – Research Trial Results and Challenges |
| Saturday, May 27, 2023 |
| 3:00 PM–3:50 PM |
| Convention Center Four Seasons Ballroom 1 |
| Area: DDA; Domain: Applied Research |
| Chair: Patrick Romani (University of Colorado, Anschutz Medical Campus) |
| CE Instructor: Nicole Tartaglia, M.D. |
| Presenting Author: NICOLE TARTAGLIA (University of Colorado School of Medicine and Children's Hospital Colorado) |
| Abstract: Advances in neuroscience have led to discovery of many potential medication targets for treatment of behavioral and cognitive disorders in autism spectrum disorder and other neurogenetic syndromes, leading to many different clinical research trials in these conditions. This presentation will introduce audiences to the different pharmaceutical trials targeting behavioral outcomes, including trials of glutamate antagonists and GABA agonists in Fragile X syndrome, and vasopressin antagonists and cannabidiol (CBD) for treatment of irritability and anxiety in autism. We will also discuss some of the challenges in designing and executing medication trials targeting behavioral outcomes in individuals with autism and other developmental disabilities, and the unmet need for strong behavioral outcome measures for use in pharmaceutical trials. |
| Instruction Level: Intermediate |
| Target Audience: The target audience includes those interested in medication treatment trials and research in autism and developmental disabilities, those involved in the development of new tools and strategies to measure behavioral outcomes in clinical trials |
| Learning Objectives: At the conclusion of the presentation, participants will be able to: (1) Understand the results of recent medication trials targeting behavior in autism, Fragile X and other neurogenetic conditions; (2) List challenges faced by investigators studying new medication treatments for behavioral and neurodevelopmental disorders; (3) Recognize important components of the tools needed to study behavioral outcomes in pharmaceutical trials |
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| NICOLE TARTAGLIA (University of Colorado School of Medicine and Children's Hospital Colorado) |
 Nicole Tartaglia, MD, MS is a Developmental-Behavioral Pediatrician and Professor of Pediatrics at University of Colorado School of Medicine and Children's Hospital Colorado. She completed her medical education and graduate studies in Clinical Investigation at University of Colorado, Pediatric training at Children's Hospital Los Angeles, and fellowship training in Developmental-Behavioral Pediatrics at the University of California Davis MIND Institute. Since 2007, she has been Director of the Denver Fragile X Clinic for children and adults affected by mutations in the FMR1 gene and Director of the eXtraordinarY Kids Clinic for individuals with sex chromosome disorders serving patients from the prenatal period to adulthood. She leads a research team focused on identifying behavioral phenotypes in these neurogenetic disorders, and conducting clinical trials for investigational medications targeting behavioral and developmental outcomes in Fragile X, autism, and sex chromosome disorders. Her clinical practice focuses on neurodevelopmental care and psychopharmacologic medication management for psychological and behavioral challenges in children and adolescents with autism, ADHD, intellectual disability and neurogenetic disorders. She lives in Colorado with her husband and 2 children, and enjoys hiking, snowboarding, backpacking, red wine, and social engagements with friends, colleagues, and family. |
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