Ways to Better Measure Neurodevelopmental Outcomes & Develop Endpoints that are Patient Centric
Time: 11:20 am
day: Day One
• Striving for clarity and certainty with measuring neurodevelopmental domains
• Cognitive development, motor development, general functional or communication, what’s the best approach?
• Developing outcome measures and clinical trial design that work in a way that makes sense and that we can use for all trials going forward.
• Finding measures that are functional for the individual
• New communication measures
• Approaches to capturing a broader assessment of clinical benefit in heterogeneous complex rare disease