Aegerion conducts both preclinical and clinical investigational studies on its product candidates. When appropriate, Aegerion may provide results of these studies to educate the research, clinical and medical communities on both the mechanisms of disease states as well as clinical results from its therapies. There are also other important scientific publications relating to the disease state and mechanism of action of our therapies from renowned authors and institutions that provide the basis for our clinical research.
Through the years, we have published and presented results relating to the efficacy and safety of our investigational therapies in full, peer-reviewed publications as well as presentations at society meetings. We have published, with our investigators, the results of our clinical trial work in world-renowned journals such as the New England Journal of Medicine(NEJM), and Nature, Clinical Practice Cardiovascular Medicine. We have presented at major society meetings such as the AHA, ADA, EAS, DALM, ISA, AALSD, and ATVB, among others. This site contains a bibliography of publications that should help educate and provide background on HoFH, FC, MTP-I and the clinical trial research of lomitapide.
Goldstein JL, Hobbs HH, Brown MS. Familial hypercholesterolemia. In: Scriver CR, Beaudet Al, Sly WS, Valle D, eds. The Metabolic and Molecular Bases of Inherited Disease. 8th ed. New York: McGraw-Hill Information Services Company; 2001:2863-913
Kolansky DM, Cuchel M, Clark BJ, et al. Longitudinal evaluation and assessment of cardiovascular disease in patients with homozygous familial hypercholesterolemia. Am J Cardiol. 2008; 102:1438-1443.
MTP and Mechanism of Action
Hussain MM, Shi J, Dreizen P. Microsomal triglyceride transfer protein and its role in apoB-lipoprotein assembly. J Lipid Res. 2003; 44:22-32.
Liao W, Hui TY, Young SG, Davis RA. Blocking microsomal triglyceride transfer protein interferes with apoB secretion without causing retention or stress in the ER. J Lipid Res. 2003; 44:978-985.
Wetterau JR, Gregg RE, Harrity TW, et al. An MTP inhibitor that normalizes atherogenic lipoprotein levels in WHHL rabbits. Science 1998;282: 751-4.
Cuchel M, Bloedon LT, Szapary PO, Kolansky DM, Wolfe ML, Sarkis A, Millar JS, Ikewaki K, Siegelman ES, Gregg RE, Rader DJ: Inhibition of microsomal triglycerides transfer protein in familial hypercholesterolemia. N Engl J Med 356: 148-156, 2007. Free Download from NEJM
Scientific Presentations and Posters
ISA 2009 PDF
Efficacy, Safety and Tolerability of the MTP--Inhibitor, AEGR--733 (Lomitapide) Combined with Atorvastatin
ATVB 2009 PDF
Microsomal Triglyceride Transfer Protein
AASLD 2009 PDF
Prevalence and Analysis of Hepatic Steatosis in Patients with Hypercholesterolemia who Lack Traditional Risk Factors Associated with Steatosis