Abstract

Background and Aims: HeFH disease severity is linked to the causative mutation type. Important mutations in HeFH include those with two variants; double heterozygous (DH) and compound heterozygous (CH), which are frequently similar phenotypically to HoFH; and functional mutations in LDLR (negative, defective and unknown). This analysis explored the efficacy of inclisiran in patients with these genotypes along with the genotypes examined previously.

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