Reflections on the Rare Drug Development Symposium

GSA Executive Director Meredith Weiss and Board Vice President Taylor Hodge recently joined patient advocates, researchers, and industry leaders in Boston for Global Genes’ 2026 RARE Drug Development Symposium. We learned so much from colleagues working toward better treatments for their own rare disease communities.

Sessions explored how natural history data can support therapeutic development, how meaningful partnerships move research forward, and how patients’ experiences can shape research from its earliest stages. Hearing about both successes and setbacks offered practical lessons for our work in Gorlin syndrome.

The experience reinforced that GSA is building the resources needed to enable discoveries that will help lead to better treatments and a cure. Our Research Roadmap provides direction, our Natural History Study helps deepen understanding of Gorlin syndrome, and our research grants and partnerships create opportunities to turn important questions into action.

Being in conversation with other rare disease organizations helps us bring context to the work we do every day. While each rare disease presents its own set of needs, constraints and opportunities, we learn a tremendous amount from each other about how to establish resources that make innovation possible, and how to sustain progress through the challenges of research and therapeutic development.