Antibody

Proprotein convertase

Kexin

Heterozygote advantage

Familial hypercholesterolemia

Monoclonal antibody

Biology

Gene

Endocrinology

Allele

Evolocumab

Cholesterol

Internal medicine

Alirocumab

Monoclonal

Immunology

Lipoprotein

Apolipoprotein A1

PCSK9

LDL receptor

Genetics

Medicine

New England Journal of Medicine

Evolocumab, a fully human monoclonal antibody directed against proprotein convertase subtilisin–kexin type 9, is widely used in adult patients to lower low-density lipoprotein (LDL) cholesterol levels. Its effects in pediatric patients with heterozygous familial hypercholesterolemia are not known.

Evolocumab in Pediatric Heterozygous Familial Hypercholesterolemia