New shot aims to slash dangerous cholesterol in teens with rare genetic disease

NCT ID NCT07473843

First seen Mar 16, 2026 · Last updated May 16, 2026 · Updated 7 times

Summary

This study tests an experimental drug called zodasiran in 12 teenagers (ages 12 to 17) with homozygous familial hypercholesterolemia, a rare genetic condition causing extremely high cholesterol. Participants receive injections of zodasiran to see if it safely lowers their LDL cholesterol over 12 months. All teens are already on maximum tolerated cholesterol-lowering treatments.

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