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Homozygous familial hypercholesterolemia

MONDO:0018328

Also known as: HoFH, homozygous familial hypercholesterolemia

23 clinical trials for this condition and its sub-types, 22 tagged with Homozygous familial hypercholesterolemia itself.

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Where it sits in the disease tree

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Part of

↑ Familial hypercholesterolemia (94)

Including sub-types (23) Tagged with Homozygous familial hypercholesterolemia (22)

Trials to join now! 10 Not yet recruiting 1 Not yet finished but already full! 6 Completed 5 Terminated 1

Experimental drug targets stubborn cholesterol in rare disease

Disease control Stopped early

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 2 • Sponsor: Arrowhead Pharmaceuticals • Aim: Disease control

Last updated Jun 27, 2026 13:06 UTC