March 18-20, 2025
Pre-Conference Workshops on March 17, 2025Sheraton Philadelphia Downtown | Philadelphia, PAExclusive insights on orphan drugs, gene therapy and rare disease from top industry experts.
Stay ahead in the evolving landscape of orphan drugs, gene therapy for rare disorders, and rare disease events with exclusive insights from industry leaders.
Speaker Interviews
Hear from top industry experts committed to shaping the future of Rare Disease. Delve into insights from the experts across the rare disease industry.
Mary McGowan & Tricha Shivas: Leading the Fight Against Sarcoidosis
Rob Frieshat: A Conversation with Uncommon Cures on Patient-Centric Strategies and Global Impact
Todd Hudson
Coming soon...
Event Insights
Hear what our attendees have to say about the Rare Disease Summit
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1000+
Clinical trials focusing on rare diseases are underway worldwide
5000+
FDA approved orphan drugs
Over 300%
Growth in the number of rare disease organisations
60+
Countries now have national rare disease plans
100+
Countries now partake in Rare Disease Day
50%
Of new drug approvals in the past 5 years have been for rare diseases
