Currently some activities focus on accelerating rare diseases drug development by using tools and methods such as real-world data, Patient Centred Outcome measures integration, streamlined gene editing and technology transfer platforms, and innovative clinical trials designs.
Learning Objectives
To identify innovative methodologies, technologies and tools for innovative therapy development; To collaborate with an innovative Rare Disease research Ecosystem and recall its funding opportunities.
Chair
Solange Corriol-Rohou, DrMed, MD, PhD
Tuesday June 17, 2025 3:00pm - 3:30pm EDT TBDWalter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
