Venglustat for treating Fabry disease

Venglustat is being developed as a treatment for Fabry disease. Fabry disease is a rare genetic condition caused by an enzyme (a type of protein) called alpha-galactosidase A. This enzyme usually breaks down a chemical called GL-3 and without it, the GL-3 builds up in various organs of the body, resulting in damage particularly in the kidneys, heart, and nervous system. Fabry disease can have a profound impact on health-related quality of life and can reduce life expectancy. Proactive management is crucial to preventing irreversible organ damage and improving long-term outcomes in people with Fabry disease. There is a need for less burdensome treatment options.