This panel will explore the vital role of patients and their families in driving innovation in rare disease therapies. By sharing personal experiences and expert insights, panelists will highlight how grassroots advocacy, research collaborations, and breakthrough technologies are transforming the landscape of drug development in rare disease. Attendees will gain a deeper understanding of the power of patient voices and actions in shaping and accelerating new rare disease treatments. Panelists will address challenges faced by rare disease communities, including access to treatments, funding hurdles, and gaps in research. Through interactive discussions and firsthand narratives, attendees will leave with practical insights into how they can engage in advocacy efforts, build meaningful partnerships with researchers and industry leaders, and contribute to the advancement of therapies that address unmet medical needs.