Meet Sue Leone!
Patient-Focused Drug Development (PFDD) meetings are one of the most powerful advocacy tools available to rare disease communities—but most patients have never heard of...
PARENTS AS RARE - EPISODE 033 Emma & Spencer - The Heart of Parents As Rare As a dad with a rare disease, I...
When Jonathan Russell lost his mother Sandra to mitochondrial disease, he and his family turned grief into action—launching what would become one of MitoAction’s...