Doctors diagnosed Patrick Girondi’s son with thalassemia in 1992, declaring he’d die by age 14. Like most other rare conditions, treatments for thalassemia were slim to none. Dr. Stanley Nelson explains how orphan disease research has progressed in the last few decades and why a cure isn’t always the goal.
- Dr. Stanley Nelson, Professor of Human Genetics, UCLA, Director of the California Center for Rare Diseases, UCLA
- Patrick Girondi, author of Flight of the Rondone, Founder, San Rocco Therapeutics