The treatments for HoFH can be quite complex and anyone with this disease is advised to be under the care of a lipid specialist. Click here
to find a lipid specialist near you.
FH management can include a combination approach – statins, cholesterol absorption inhibitors, and bile acid sequestrants. Often, HoFH patients will require even more intensive therapy to bring their LDL cholesterol (“bad cholesterol”) levels down.
– LDL apheresis (also called lipoprotein apheresis, LA) can be performed on HoFH patients as well as more serious HeFH patients. This procedure involves blood cleansing every other week (or sometimes weekly) with a special machine that looks like a dialysis machine. The procedure takes between one and a half to three hours, and typically drops LDL levels by 70%. Unfortunately, LDL rises over time and, for this reason, repeat treatments are necessary. About 60 lipoprotein apheresis centers exist in the US. To locate a center near you, click here
Lomitapide (marketed as Juxtapid in the U.S.)
– Lomitapide works in the liver by partially blocking the production of LDL particles. It is approved as an adjunct to a low-fat diet and other lipid lowering treatments. It is an oral capsule.
Mipomersen (marketed as Kynamro in the U.S.)
-Mipomersen is an antisense drug that leads to the decline of apoB RNA in the liver (apoB is necessary for the formation of LDL particles). It is administered as a weekly injection.
Additional therapies are in clinical trials and development.