Congratulations to first author, Johnathan Seth Parham, MD and senior author, Anne Carol Goldberg, MD on their recent publication, “Mipomersen and its use in familial hypercholesterolemia” which was published online December 10 in Expert Opinion on Pharmacotherapy.
The authors wrote in their conclusion, “The armamentarium available to the clinician treating HoFH includes multiple medications. Because several of the most potent medications, such as statins and PCSK9 monoclonal antibodies work by increasing LDL receptor function, most patients with HoFH do not achieve recommended LDL-C targets. For these patients, additional treatment regimens, including mipomersen, which target different pharmacologic mechanisms, should be considered as adjunct therapy in patients who do not meet LDL-C goals and remain at high risk for cardiovascular disease. In addition, adverse effects, particularly hepatotoxicity, local site injection reactions, and concern for hepatic steatosis with long-term use may limit use significantly.”