The Power of a Rare Disease Community

What can families do to move the needle on research for their child's condition — and how much does it actually matter? Pangkong Fox, PhD, knows this question from both sides: as a cell and molecular biologist, and as mom to Alex, diagnosed with a CACNA1A-related disorder in 2021. Now the Science Engagement Director for the CACNA1A Foundation, she uses "conversational science" to help parents become powerful advocates and active partners in the search for treatments. In this opening keynote, she explores what rare disease communities — including ours — are uniquely positioned to accomplish together.