Patients and families are at the heart of every rare disease journey. Guided by their experiences, we are committed to transforming care in rare disease and advancing meaningful innovation where unmet needs remain.
Across rare disease, patients continue to navigate serious conditions with few effective treatments. Hear from Renee Gala, President and CEO, on how we are sharpening our strategic focus to drive innovation and strengthen our commitment to delivering meaningful impact for patients and their families. Armando’s journey with cholangiocarcinoma (CCA), a rare biliary tract cancer, reflects the power of connection and resilience. Throughout his experience, he shared how the support of loved ones brought him strength and peace. His words continue to inspire the cancer community, honoring the courage and spirit he carried into every conversation. Lisa shares the story of her son Jace, who faced his diagnosis of a rare brain cancer with courage and compassion. His outlook continues to inspire the support Lisa now offers to other families navigating childhood cancer.