EP 40 Maureen Smith

How can rare disease research become more human, more effective, and more accountable to the people it’s meant to serve? In this powerful episode of Rare Awareness Radio, host Richard Juknavorian speaks with Maureen Smith, a lifelong rare disease advocate whose journey began as a child participant in clinical trials and evolved into decades of leadership in patient-partnered research. From her own lived experience with an ultra-rare condition to her national work advancing research collaboration, Maureen offers a compelling vision for the future of rare disease innovation. Together, Richard and Maureen explore why patients must be more than research subjects—they must be true partners in study design, governance, clinical trials, and outcome measurement. Maureen shares lessons from her work with CANPKU+, including the development of registries, core outcome sets, youth engagement initiatives, and strategies to reduce power imbalances between institutions and families. This conversation also tackles urgent questions about funding, tokenism, transparency, and how research priorities should be driven by real, unmet needs—not by academic curiosity alone. Maureen’s insights are practical, passionate, and deeply earned. If you care about patient-centered healthcare, rare disease advocacy, clinical research reform, or the power of lived experience, this is an episode you won’t want to miss. More on CANPKU+: https://canpku.org/ More on Rare Awareness Radio: https://rareawarenessradio.org/ #RareAwarenessRadio #RareDisease #PatientAdvocacy #ClinicalResearch #RareDiseaseResearch #PatientEngagement #PKU #CANPKU #HealthcareInnovation #ResearchPartnerships