Welcome to the EAS 2023 Interactive Program

Latest developments and real-world experience in the management of homozygous familial hypercholesterolaemia - Industry Sponsored Session by Ultragenyx. This symposium will focus on the clinical management of homozygous familial hypercholesterolaemia (HoFH), a rare genetic disorder characterized by extremely high plasma low-density lipoprotein cholesterol (LDL-C) concentrations. Left untreated, HoFH can have devastating consequences such as premature coronary heart disease and death before age 20 years. The first presentation will provide an overview of HoFH, including its aetiology, clinical presentation, diagnosis, comorbidities and the goals of treatment, and will highlight the limitations of conventional therapy (statins and cholesterol absorption inhibitors) for lowering LDL-C in patients with HoFH. The second presentation will summarize the clinical data for commonly prescribed add-on therapies (such as inhibitors of proprotein convertase subtilisin/kexin 9 or microsomal transfer protein) as well as a novel antibody therapy targeting angiopoietin-like protein 3 (ANGPTL3), which is expected to be available to clinicians in Europe in 2023. The strengths and any important considerations applicable to the management of patients for each add-on therapeutic agent will also be considered. Finally, the third presentation will provide real-world clinical experience of anti-ANGPTL3 antibody therapy for the treatment of HoFH via a series of case studies in both adult (aged ≥18 years) and adolescent (aged ≥12 to 17 years) patient populations.