In the pilot episode of Gene Pool Media’s newest podcast Rare Rebels, parent Mark Dant shares how grassroots advocacy and a scientist’s persistence reshaped rare disease drug development.
All tagged mucopolysaccharidosis
#372 Fighting for Time: Hunter Syndrome, Gene Therapy & Urgent Advocacy
Kristin McKay, Executive Director of Project Alive, is dedicated to accelerating treatments, expanding care, and supporting families affected by Hunter Syndrome, a disease that impacted her brother and now her son.