Meet Stephanie Harry!
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...
Tune in to meet Lukas from Probably Genetic and learn how you can find out if their DNA test is right for you!
Walker, Madison, and Jordan are three relentless advocates whose determination helped push a life-changing therapy for Barth syndrome across the finish line. In this...