- Fabry disease involves multiple organ systems in the body. It is due to storage of a complex molecule that we will call GL3 or related molecules. Because they cannot be broken down, they build up in the body. And as the cells get more and more of the GL3, that impacts the cellular functions. And the differences in the cell functions varies depending on the organ system that you're talking about.

Storage can be detected starting in the prenatal period. It then progresses and becomes symptomatic, in classical Fabry Disease in childhood, becoming more severe going into adolescence and adulthood. And you start to see potentially serious or life-threatening complications going into mid-20s and beyond.