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Vasc Health Risk Manag ; 18: 73-80, 2022.
Artigo em Inglês | MEDLINE | ID: mdl-35221690

RESUMO

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.


Assuntos
Anticolesterolemiantes , Hiperlipoproteinemia Tipo II , Anticolesterolemiantes/efeitos adversos , Humanos , Hiperlipoproteinemia Tipo II/diagnóstico , Hiperlipoproteinemia Tipo II/tratamento farmacológico , Hiperlipoproteinemia Tipo II/genética , Oligonucleotídeos , Medidas de Resultados Relatados pelo Paciente , Qualidade de Vida
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