WinQure Medicine recently participated in COMBINEDBrain’s Research Roundtable on Rare Genetic Neurodevelopmental Disorders (RR-RGND), bringing together patient advocacy groups, researchers, industry representatives, and the FDA to discuss how therapies for rare genetic diseases can reach patients faster.
The meeting highlighted the FDA’s vision for the future of rare disease drug development and the determination to turn that vision into practical change. Discussions focused on rethinking existing approaches and finding ways for regulators, researchers, industry, and patient communities to work together more effectively.
“It was remarkable to hear the FDA speak with such a strong vision for the future of rare disease and such determination to make that vision a reality. Everyone in the room was united around the same goal: to rethink how we get therapies to patients faster. I left feeling like change is here,” said Dr. Stephanie Manton, CEO and Co-founder of WinQure Medicine.
WinQure Medicine thanks Dr. Terry Jo Bichell and the rest of the COMBINEDBrain team for bringing together the people working to shape the future of rare disease drug development.