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Sign up to view this video Join Now Closing Panel, • 9/22/25 Closing Panel: Beyond “One Disease At a Time”: Platform Approaches to Accelerate Gene Therapy Trials You Might Also Like Innovating Clinical Outcome Assessments in Rare Disease Trials: Integrating Patient-Centered Measures and Digital Health Technologies Beyond the Feasibility Survey: Strategic Site Selection to Accelerate Trials and Optimize Resources Reducing Patient Burden in Rare Disease Trials Optimizing CRO and Vendor Selection - Examples from the Field Beyond Biotech: The Rise of Nontraditional Sponsors in Rare Disease Trials