HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA (HOFH)
Clinical trials for HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA (HOFH) explained in plain language.
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Rare cholesterol drug may cut heart attacks in genetic disorder
Disease control OngoingThis study looks at whether lomitapide, a drug that lowers very high cholesterol, can reduce serious heart problems like heart attacks and strokes in people with a rare genetic condition called homozygous familial hypercholesterolemia (HoFH). Researchers will compare heart events…
Matched conditions: HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA (HOFH)
Sponsor: Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi) • Aim: Disease control
Last updated May 26, 2026 10:54 UTC
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New RNAi drug shows promise for rare, severe high cholesterol
Disease control OngoingThis study tests an experimental RNAi drug called VSA003 in 46 Chinese adolescents and adults with homozygous familial hypercholesterolemia (HoFH), a rare genetic condition causing extremely high cholesterol and early heart disease. The drug aims to lower harmful cholesterol by b…
Matched conditions: HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA (HOFH)
Phase: PHASE3 • Sponsor: Visirna Therapeutics HK Limited • Aim: Disease control
Last updated May 26, 2026 10:51 UTC