Publications

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.

HoFH

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.

Lomitapide

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 Michael Davidson1, Thomas Littlejohn2, Sheila Rodstein3, Carol Price4, Matthew Parris4, William Sasiela4, Daniel Rader5 1Radiant Research, Chicago, IL; 2Piedmont Medical Research, Winston-Salem, NC; 3Radiant Research, Minneapolis, MN; 4Aegerion Pharmaceuticals, Bridgewater, NJ; 5University of Pennsylvania, Philadelphia, PA
	ATVB 2009 PDF Microsomal Triglyceride Transfer Protein Jeffrey Billheimer*, Debra Cromley*, William Sasiela^ and Daniel J. Rader* *University of Pennsylvania, Institute for Translational Medicine and Therapeutics, Philadelphia, PA ^ Aegerion Pharmaceuticals, Bridgewater, NJ
	AASLD 2009 PDF Prevalence and Analysis of Hepatic Steatosis in Patients with Hypercholesterolemia who Lack Traditional Risk Factors Associated with Steatosis Scott Grundy, Michael Davidson, Harold Bays, Mark Rosen, Emma Meagher, LeAnne Bloedon, Carol Price, Matthew Parris, William Sasiela, Daniel Rader From The University of Texas Southwestern, Dallas, TX (SG); Radiant Research, Chicago, IL (MD); Louisville Metabolic and Atherosclerosis Research Center, Louisville, KY (HB); University of Pennsylvania, Philadelphia, PA (MR, EM, LB, DR), and Aegerion Pharmaceuticals, Bridgewater, NJ (LB, CP, MP, WS)