Leveraging Natural History Studies in Clinical Trial Design for Rare CNS and Neurodevelopmental Disorders

Time: 10:30 am
day: Day Two


  • Despite common traits and symptoms, each NDD displays a high variability in their phenotype (e.g. ASD, Down syndrome, Rett syndrome, CDD)
  • Characterizing this disease variability together with potential endpoints evaluation is critical before embarking into drug development
  • Exploring case studies of how observational / natural history studies in NDD helped to improve early and late stage clinical trials