The Translational Neuroscience Center seeks to improve the lives of children and adolescents with nervous system disorders by tackling conditions that once seemed resistant to therapy, such as autism spectrum disorder, muscular dystrophy, and brain tumors. The center provides cutting edge tools and facilities to aid discovery. The Repository Core for Neurological Disorders, for example, is a database and biological specimen bank of high-quality clinical data with paired DNA and protein samples from patients. These data support new translational research, clinical trials, and enhanced collaborations among users. The center’s efforts directly impact clinical care, as is the case with the CDKL5 Clinic, which is helping clinicians understand this ultra-rare disorder’s features and trajectory over time.