Heterozygous Familial Hypercholesterolemia research hub

A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease

This study is testing a new gene-editing treatment called VERVE-102 for people with high cholesterol due to a genetic condition (Familial Hypercholesterolemia) or early heart disease. It aims to see if VERVE-102 is safe and can help lower 'bad' cholesterol.

Randomized, Placebo-Controlled, Double-Blind, Phase 3b Study to Evaluate the Efficacy and Safety of Lerodalcibep in Children and Adolescents, 6 to 17 Years of Age, with Heterozygous Familial Hypercholesterolemia on Stable Diet and Oral Lipid-Lowering Therapy (LIBerate-Kids)

This study looks at a new medicine called lerodalcibep for children and teenagers aged 6 to 17 with a genetic condition causing high cholesterol (Heterozygous Familial Hypercholesterolemia). It aims to see if the medicine can lower their cholesterol safely and effectively, compared to a dummy medicine.

Long-term Follow-up of Participants Dosed With an Investigational Gene Editing Therapy for Cardiovascular Disease

This study is following people who've had an experimental gene therapy for certain heart conditions. It checks how the treatment affects them over a long time, up to 15 years, looking for any lasting benefits or side effects to make sure it's safe and works well.

Study 1002-041: An Open-Label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Bempedoic Acid in Pediatric Patients (6 to 17 Years of Age) with Heterozygous Familial Hypercholesterolemia