Closing Panel, • 9/22/25 Closing Panel: Beyond “One Disease At a Time”: Platform Approaches to Accelerate Gene Therapy Trials You Might Also Like Real-World Evidence That Matters: A Hands-On Workshop in Building Credible Value Stories Opening Keynote: The Future of Rare Disease Trials Designing Trials with the End in Mind: Building Market Access into Rare Disease Clinical Development Purpose-Driven Metrics: Running Gene Therapy Trials with KPIs That Matter Patient Recruitment and Engagement in in Global, Multisite Rare Disease Trials
Closing Panel, • 9/22/25 Closing Panel: Beyond “One Disease At a Time”: Platform Approaches to Accelerate Gene Therapy Trials You Might Also Like Real-World Evidence That Matters: A Hands-On Workshop in Building Credible Value Stories Opening Keynote: The Future of Rare Disease Trials Designing Trials with the End in Mind: Building Market Access into Rare Disease Clinical Development Purpose-Driven Metrics: Running Gene Therapy Trials with KPIs That Matter Patient Recruitment and Engagement in in Global, Multisite Rare Disease Trials
