Why the study?
Does mipomersen impact health-related quality of life and patient-reported outcomes in patients with familial hypercholesterolemia?
Does mipomersen impact health-related quality of life and patient-reported outcomes in patients with familial hypercholesterolemia?
This review summarizes the mechanism, evidence, limitations, and impact on health-related quality of life of mipomersen for homozygous familial hypercholesterolemia.
Mipomersen may offer adjunctive LDL-C lowering in FH; leaves open long-term CV outcomes and safety.
Familial hypercholesterolemia (FH) is an autosomal dominant condition that leads to significantly elevated low-density lipoprotein cholesterol (LDL-C) levels and an elevated risk for cardiovascular disease. Mipomersen is an antisense oligonucleotide inhibitor targeted to apolipoprotein B-100 (apoB-100) mRNA that is administered via subcutaneous injection. Once administered, mipomersen causes selective degradation of the apoB-100 mRNA and inhibition of protein translation. This ultimately results in substantial reductions in LDL-C and other lipoprotein levels. Mipomersen is approved for the treatment of homozygous FH. In this review, we discuss its mechanism, current evidence, limitations of use including adverse events, and impact on health-related quality of life.
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