Changing How We Target Rare Diseases

By Lindsey Sutton & Melissa Goetz

When we founded the FCS Foundation in 2016, one of our main goals was to help more people learn about and understand the impact of familial chylomicronemia syndrome (FCS), a rare disorder where the body is unable to break down triglycerides (fats) in the body. FCS affects only about one in a million people, and so most people, including many clinicians, are not familiar with it. As we worked to build awareness, we also quickly noticed that even leaders in industry and at the FDA had a limited understanding of this disease.