On Medicine The impact of treatment access on well-being of patients living with a rare disease: reflections from lived experiences of patients with Familial Chylomicronemia Syndrome

Unfortunately, 48% of FCS patients report being misdiagnosed (3). Even with adherence to extremely restricted diet, patients continue to experience pancreatitis and a reduced quality of life (3,5), making patients feel defeated. Glybera, the world’s first approved gene therapy, priced at a million dollars was approved for FCS in Europe. “The FDA seems to have very little understanding of what FCS patients deal with on daily basis. “All they kept looking at were the number of pancreatitis attacks and hospitalizations” several patients shared.