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17215532 10.1056/NEJMoa061189 1:CAS:528:DC%2BD2sXksVKmtQ%3D%3D
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23122768 10.1016/S0140-6736(12)61731-0 1:CAS:528:DC%2BC38Xhs1ajsb%2FM This study showed that lomitapide, a microsomal triglyceride transfer protein inhibitor, produces significant reductions in LDL cholesterol in patients with homozygous familial hypercholesterolemia, and found that elevations in transaminase levels could be corrected with dose reduction or temporary cessation of the medication. These findings help to establish a role for the use of lomitapide in clinical practice, and provide guidance for addressing the potential side effect of transaminase elevation
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Cuchel M, Meagher EA, du Toit Theron H, Blom DJ, Marais AD, Hegele RA, et al. Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 study. Lancet. 2013;381:40-6. This study showed that lomitapide, a microsomal triglyceride transfer protein inhibitor, produces significant reductions in LDL cholesterol in patients with homozygous familial hypercholesterolemia, and found that elevations in transaminase levels could be corrected with dose reduction or temporary cessation of the medication. These findings help to establish a role for the use of lomitapide in clinical practice, and provide guidance for addressing the potential side effect of transaminase elevation.
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Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous famililal hypercholesterolaemia: A randomised, double-blind, placebo-controlled trial
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20227758 10.1016/S0140-6736(10)60284-X 1:CAS:528:DC%2BC3cXjs1ait74%3D This study demonstrated that mipomersen, an apolipoprotein B synthesis inhibitor, has beneficial effects on the lipid profile of patients with homozygous familial hypercholesterolemia, including a reduction in LDL cholesterol, and that it is generally safe to use
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Raal FJ, Santos RD, Blom DJ, Marais AD, Charng M, Cromwell WC, et al. Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous famililal hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial. Lancet. 2010;375:998-1006. This study demonstrated that mipomersen, an apolipoprotein B synthesis inhibitor, has beneficial effects on the lipid profile of patients with homozygous familial hypercholesterolemia, including a reduction in LDL cholesterol, and that it is generally safe to use.
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Barter PJ, Caulfield, M.; Eriksson, M.; Grundy, S. M.; Kastelein, J. J. P.; Komajda, M.; Lopez-Sendon, J.; Mosca, L.; Tardif, J.; Waters, D. D.; Shear, C. L.; Revkin, J. H.; Buhr, K. A.; Fisher, M. R.; Tall, A. R.; and B. Brewer Effects of Torcetrapib in Patients at High Risk for Coronary Events. N Engl J Med 2007;357.
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Kastelein JJP, van Leuven S. I.; Burgess, L.; Evans, G. W.; Kuivenhoven, J. A.; Barter, P. J.; Revkin, J. H.; Grobbee, D. E.; Riley, W. A.; Shear, C. L.; Duggan, W. T.; and M. L. Bots. Effect of Torcetrapib on Carotid Atherosclerosis in Familial Hypercholesterolemia. N Engl J Med 2007;356.
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Cannon CP, Shah, S.; Dansky, H. M.; Davidson, M.; Brinton, E. A.; Gotto, A. M.; Stepanavage, M.; Xueyu Liu, S.; Gibbons, P.; Ashraf, T. B.; Zafarino, J.; Mitchel, Y.; Barter, P. Safety of Anacetrapib in Patients with or at High Risk for Coronary Heart Disease. N Engl J Med 2010;363.
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Stein EA, Mellis, S.; Yancopoulos, G. D.; Stahl, N.; Logan, D.; Smith, W. B.; Lisbon, E.; Gutierrez, M.; Webb, C.; Wu, R.; Du, Y.; Kranz, T.; Gasparino, E.; and G. D. Swergold. Effect of a monoclonal antibody to PCSK9 on LDL cholesterol. N Engl J Med 2012;366.
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22463922 10.1016/j.jacc.2012.03.007 1:CAS:528:DC%2BC38XovV2ntLs%3D This study showed that SAR236553, a monoclonal antibody against PCSK9, was generally well-tolerated and reduced LDL cholesterol in a dose and frequency dependent manner in patients on atorvastatin, but with baseline LDL cholesterol levels still ≥ 100 mg/dl. Although this was not a study of the treatment of familial hypercholesterolemia, the significance of these findings suggests that antibodies against PCSK9 should be tested further and considered among the potential emerging therapies for familial hypercholesterolemia
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McKenney JM, Koren MJ, Kereiakes DJ, Hanotin C, Ferrand AC, Stein EA. Safety and efficacy of a monoclonal antibody to proprotein convertase subtilisin/kexin type 9 serine protease, SAR236553/REGN727, in patients with primary hypercholesterolemia receiving ongoing stable atorvastatin therapy. J Am Coll Cardiol. 2012;59:2344-53. This study showed that SAR236553, a monoclonal antibody against PCSK9, was generally well-tolerated and reduced LDL cholesterol in a dose and frequency dependent manner in patients on atorvastatin, but with baseline LDL cholesterol levels still ≥ 100 mg/dl. Although this was not a study of the treatment of familial hypercholesterolemia, the significance of these findings suggests that antibodies against PCSK9 should be tested further and considered among the potential emerging therapies for familial hypercholesterolemia.
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