Organizer
CNS Research Committee
Daniel Calame, MD, PhD
Baylor College of Medicine
Description
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.