What Are the Clinical Implications?
LDL-C reduction
GENETIC DISEASE, HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA, HOFH, HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA
Although this study did not show a statistically significant reduction in LDL-C level in patients with homozygous familial hypercholesterolemia, inclisiran demonstrated a sustained reduction in PCSK9 levels and was well-tolerated, with a safety profile consistent with previous studies.Despite the lack of significant LDL-C reduction with inclisiran, the observations in the subsets with some residual 
Homozygous familial hypercholesterolemia (HoFH) is a rare genetic disease that affects ≈1 in 300 000 people worldwide.Lowering LDL-C levels in patients with HoFH reduces the incidence of cardiovascular eventsThe phase 2 ORION-2 (URL: 