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Familial hypercholesterolemia

MONDO:0005439

An inheritable form of hyperlipidemia, in which there are excess lipids in the blood.

Also known as: hyperlipoproteinemia type II, type II hyperlipidemia

120 clinical trials for this condition and its sub-types.

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Sub-types

Homozygous familial hypercholesterolemia (21) Hypercholesterolemia, familial, 1 (18) Hypercholesterolemia, autosomal dominant, 3 (3) Hypercholesterolemia, autosomal dominant, type B (1) Hypercholesterolemia due to cholesterol 7alpha-hydroxylase deficiency (0)

Broader categories

Disease (618) Hyperlipidemia (264) Metabolic disease (215) Hereditary disease (172) Inherited lipid metabolism disorder (165) Inborn errors of metabolism (42) Human disease (14) Hyperlipoproteinemia (7) Familial hyperlipidemia (5) Disease of genetic or genomic mechanism (2)

Trials to join now! 48 Not yet recruiting 17 Not yet finished but already full! 19 Completed 35 Terminated 1

Experimental drug targets stubborn cholesterol in rare disease

Disease control Terminated

This study tested an experimental drug called ARO-ANG3 in 18 people with homozygous familial hypercholesterolemia (HoFH), a rare genetic condition causing extremely high cholesterol. Participants received injections of the drug and were monitored for safety and cholesterol change…

Phase: PHASE2 • Sponsor: Arrowhead Pharmaceuticals • Aim: Disease control

Last updated Jun 27, 2026 13:06 UTC