Gene Discovery to Clinical Trials: How Clinical and Basic Research Have Intersected to Develop and Test New Therapies for Rett Syndrome
Jeffrey Neul is director of the Vanderbilt Kennedy Center and the Annette Shaffer Eskind Professor of Pediatrics at Vanderbilt University Medical Center. Neul’s research is focused on Rett syndrome and related genetically based neurodevelopmental disorders. It integrates laboratory-based exploration of disease mechanisms, biomarker discovery and preclinical therapy evaluation with clinical research including longitudinal natural history…