Keynote topics include:
- Science Spotlight – Advancements in Gene Therapy that are Tearing Down Barriers to Discovery in Rare Disease;
- Regulatory Update – Program Updates and New Initiatives within the FDAʼs Orphan Drug Program;
- Technology Showcase – How A.I. and Advanced Data Analytics are Reshaping Complex Diagnostics and Decision-Making;
- Patient Perspectives – Cures, Community Collaboration and the Care Continuum for Patients and Caregivers; and
- Funding Focus – The Impact of Disruptive Investors and Insight on What Investors Look For and Financial Models for Success.