WASHINGTON, May 23, 2025 –The National Organization for Rare Disorders (NORD®) will convene leading voices in science, industry, and regulation at its inaugural Rare Disease Scientific Symposium, a two-day conference focused on accelerating translational and clinical research for rare diseases. The event will take place June 2-3 in Washington, D.C.
Vinay Prasad, MD, MPH, the newly appointed Director of the U.S. Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER), will deliver the keynote address. The Symposium will feature insights from more than two dozen world-renowned experts representing top academic institutions, biopharmaceutical companies, and government agencies, including the FDA and the National Institutes of Health (NIH).
“For more than 40 years, NORD has driven progress in rare disease science by fostering collaboration,” said Pamela K. Gavin, NORD Chief Executive Officer. “At a time of growing uncertainty in research, the NORD Rare Disease Scientific Symposium will serve as a vital platform for uniting scientific expertise, regulatory insight, and patient-centered perspectives to shape the future of rare disease treatment.”
With over 10,000 known rare diseases – and 95% lacking an FDA-approved therapy – the need for breakthroughs has never been more urgent. Yet researchers face major hurdles: small patient populations, limited datasets, and complex and often misunderstood conditions.
“These challenges can seem overwhelming when tackled in isolation,” said Tracey Sikora, NORD Vice President of Clinical and Research Programs. “But when researchers, clinicians, and patient advocates collaborate, progress accelerates. This Symposium is built on that core belief: that partnerships are essential to advancing science and improving lives.”
The program was shaped with guidance from a 2024 report by the National Academies of Sciences, Engineering, and Medicine, which emphasized the need for stronger engagement between the FDA and rare disease communities, increased regulatory flexibility, and the integration of patient-driven data into research and development.
Symposium highlights include:
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Keynote address by Vinay Prasad, MD, MPH, the newly appointed FDA CBER Director.
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More than 20 expert-led sessions exploring innovative study designs for small populations, real-world and patient-reported data in research, and cross-sector collaboration strategies.
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Speakers from the FDA, NIH, European Medicines Agency, leading pharmaceutical and biotech companies, and more than a dozen NORD® Rare Disease Centers of Excellence, a network of over 60 premier medical institutions advancing diagnosis, treatment, and research for rare disorders.
Registration is now open at NORD Scientific Symposium – Advancing Innovation in Rare Disease Research, where the full agenda and speaker lineup are also available.