HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA (HOFH)
Clinical trials for HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA (HOFH) explained in plain language.
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Rare gene disorder drug may slash heart attack risk
Disease control OngoingThis study looks at whether lomitapide, a medication that lowers extremely high cholesterol, can reduce major heart problems like heart attacks and strokes in people with homozygous familial hypercholesterolemia (HoFH). Researchers will compare heart events in the three years bef…
Matched conditions: HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA (HOFH)
Sponsor: Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi) • Aim: Disease control
Last updated May 17, 2026 05:37 UTC
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New shot could slash Sky-High cholesterol in rare genetic disease
Disease control OngoingThis study tests a new medicine called VSA003 in 46 Chinese teenagers and adults with homozygous familial hypercholesterolemia (HoFH), a genetic condition causing extremely high cholesterol and early heart disease. VSA003 uses RNAi technology to lower a protein (ANGPTL3) that con…
Matched conditions: HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA (HOFH)
Phase: PHASE3 • Sponsor: Visirna Therapeutics HK Limited • Aim: Disease control
Last updated May 15, 2026 11:54 UTC