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Updated: Mar 24, 2026

A Familial Hypercholesterolemia Human Liver Chimeric Mouse Model Using Induced Pluripotent Stem Cell-derived Hepatocytes
Published on: September 15, 2018
Lomitapide for use in patients with homozygous familial hypercholesterolemia: a narrative review
Daniela Neef1, Heiner K Berthold2, Ioanna Gouni-Berthold1
1a Center of Endocrinology, Diabetes and Preventive Medicine (ZEDP) , University of Cologne , Cologne , Germany.
Abstract:
Lomitapide is a drug recently approved for the treatment of patients with homozygous familial hypercholesterolemia. In this article we discuss briefly the pharmacology of this drug followed by a comprehensive narrative review of the available preclinical and clinical data on its safety and efficacy. Only data published as full papers are presented, with the exception of one long-term open-label extension study, which is available only in abstract form.
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