FIrst interim results of the global, longitudinal, pharmaco-epidemiologic, observational registry on gene therapy in the management of lipoprotein lipase deficiency (geniall)

Abstract

Aim: Background: Lipoprotein Lipase Deficiency (LPLD) is a rare genetic disease resulting in chylomicronemia and increased risk of pancreatitis. Alipogene tiparvovec (Glybera®) is a gene therapy approved for treatment of LPLD that decreases the incidence and severity of pancreatitis for up to 6 years. A 15-year, international, multicenter, observational Registry was launched in 2014 to collect further data on epidemiology of LPLD and to continue the evaluation of long-term safety and clinical response to gene therapy.