1. Therapy for severe FH:
· Pharmacotherapy – lipid-modifying drugs
· Extracorporeal removal of LDL-apheresis
· Surgical therapy – portacaval shunt, partial bypass
· Methods to restore LDL receptor activity – liver transplantation, gene therapy.
2. Despite the absence of RCTs in subjects with FH, since their introduction in the late 1980s, statins have become the mainstay of therapy. While statins and ezetimibe constitute first-line therapy, they provide less than optimal LDL-C reduction in severe FH.
3. Homozygous FH mainly requires combination therapy: high-dose statin, ezetimibe, with or without apheresis.
Professor Raal is currently Professor and Head of the Division of Endocrinology and Metabolism, as well as Director of the Carbohydrate and Lipid Metabolism Research Unit, University of the Witwatersrand, Johannesburg, South Africa. Prof frederick raal is a Frederick J. Raal, MBBCh, FRCP, FCP(SA), Cert Endo, MMED, PhD Head, Division of Endocrinology & Metabolism Director, Carbohydrate and Lipid Metabolism Research Unit Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa.
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