Bringing CNS Members Together to Make Children’s Lives Better



CNS Research Committee

Daniel Calame, MD, PhD
Baylor College of Medicine


Dr. Goodspeed will discuss the challenges and opportunities in targeted drug development in rare genetic neurodevelopmental disorders. This will include discussions around patient identification, variant interpretation, natural history studies, biomarker development, and eventually different clinical trial designs that can serve smaller patient populations using some real-world examples. Dr. Goodspeed will also discuss the considerations around gene-based therapies such as gene transfer therapy and anti-sense oligonucleotides.

About the Speakers

Kimberly D. Goodspeed, MD

Kimberly D. Goodspeed, MD

Kimberly D. Goodspeed, MD
Assistant Professor of Pediatrics, Division of Child Neurology
Secondary appointments in Neurology & Psychiatry
Center for Autism Care – Neurodevelopmental Disabilities Specialist
Dedman Family Scholar in Clinical Care

Dr. Goodspeed is a Child Neurologist and Neurodevelopmental Specialist focused on caring for patients and families with autism spectrum disorder and intellectual disability. Her research focus is on clinical trial readiness and biomarker development in rare genetic neurodevelopmental disorders by working with patient communities to eventually bring targeted therapies to patients who have very few options. She is currently an Assistant Professor of Pediatrics in the Division of Child Neurology at the University of Texas Southwestern Medical Center. She cares for patients and conducts clinical and translational research at the UT Southwestern and Children’s Health Dallas Center for Autism Care.