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