Utilizing Natural History Data for Identifying Best Clinical Outcomes

Time: 11:00 am
day: Day Two


  • Access to Natural History data from longitudinal studies increases our knowledge of rare and genetic neurodegenerative disease onset and progression
  • Involvement of people living with rare & genetic neurodegenerative disease or their carer is key to selecting most meaningful symptoms
  • Development of new clinical outcomes requires early collaboration between academics, pharma and regulators but remains very challenging at prodromal/early stage of the disease with absence or low functional impact