THERAPY: PCSK9 inhibitors for treating familial hypercholesterolaemia

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Abstract

Familial hypercholesterolaemia is caused by mutations in genes that code for proteins involved in cholesterol metabolism. Patients heterozygous for mutations inLDLRrespond to statin treatment, whereas individuals with homozygousLDLRmutations do not. PCSK9 inhibitors have been developed for treating familial hypercholesterolaemia, and results are promising for patients with either heterozygous or homozygous familial hypercholesterolaemia.

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