Anti-ANGPTL3 Antibody SHR-1918 for Homozygous Familial Hypercholesterolemia: A Nonrandomized Clinical Trial
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D Peng, L Wang, L Pi, Y Xu, J Chen, Y Wu…
JAMA …, 2026
jamanetwork.com
Importance Homozygous familial hypercholesterolemia (HoFH) is a rare, life-threatening genetic disorder. Patients with HoFH have markedly elevated low-density lipoprotein cholesterol (LDL-C) levels from birth, and their activity of LDL receptor (LDLR) is typically absent or severely impaired. However, efficacy of traditional lipid-regulating agents relies on residual LDLR function. Angiopoietinlike 3 (ANGPTL3)–directed therapies could reduce lipid levels through an LDLR-independent pathway. Objective To evaluate SHR-1918, a fully …
