Mipomersen (Kynamro)

概览

Mipomersen is an oligonucleotide drug used for the treatment of homozygous familial hypercholesterolemia.

作用机制

Mipomersen binds to the mRNA that codes for apoB-100. This binding leads to double-stranded RNA, which is degraded by RNase H and prevents translation of the mRNA to form the apo B-100 protein.

适应症

Used in patients with homozygous familial hypercholesterolemia as an adjunct to diet and other lipid-lowering medications.

• Homozygous Familial Hypercholesterolaemia (HoFH)