DTRF Executive Director, Katie Doyle Myers, recently joined fellow rare disease advocates at the NORD Breakthrough Summit, connecting with leaders across the rare disease community and exploring ways to strengthen research, collaboration, and patient care.
Several themes stood out as especially relevant to our work:
- Patient registries can drive discovery by helping researchers better understand lived experience, unmet needs, and real-world outcomes.
- What matters to patients should help define progress, including quality of life, treatment burden, and daily functioning.
- Rare diseases require tailored approaches to clinical trials, regulatory decision-making, and treatment development.
- Patient advocacy organizations are essential research partners, helping build the communities, relationships, and infrastructure that make progress possible.
- One core message came through especially clearly: people living with rare diseases hold invaluable knowledge, and their experiences must help shape the future of research and care.
We are grateful to NORD and the many leaders who shared their expertise. Learn more about NORD: https://rarediseases.org/
