Exclusive 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.
Discover how collaboration is revolutionizing rare disease care in 2026.
From navigating complex policy reforms to harnessing AI responsibly, the Rare Disease Summit 2026 showcases how patients, policymakers, clinicians, and industry leaders are uniting to transform challenges into breakthrough opportunities. Explore five critical issues, from data scarcity to cross-functional alignment, and learn how strategic partnerships are accelerating diagnosis, improving access, and delivering real impact for rare disease communities worldwide.
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
Justin Bush: Shaping the Future of HAE Care Through Innovation
Event Insights
Hear what our attendees have to say about the Rare Disease Summit
Find Your Perfect Rare Disease Summit Experience!
Meet the Leaders Council
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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
