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Pediatric experience with mipomersen as adjunctive therapy for homozygous familial hypercholesterolemia  Frederick J. Raal, PhD, Marjet J. Braamskamp,

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Presentation on theme: "Pediatric experience with mipomersen as adjunctive therapy for homozygous familial hypercholesterolemia  Frederick J. Raal, PhD, Marjet J. Braamskamp,"— Presentation transcript:

1 Pediatric experience with mipomersen as adjunctive therapy for homozygous familial hypercholesterolemia  Frederick J. Raal, PhD, Marjet J. Braamskamp, MD, PhD, Sheryl L. Selvey, PharmD, Charlotte H. Sensinger, MA, John J. Kastelein, MD, PhD  Journal of Clinical Lipidology  Volume 10, Issue 4, Pages (July 2016) DOI: /j.jacl Copyright © 2016 Sanofi-Genzyme Terms and Conditions

2 Figure 1 Mipomersen mechanism of action. apo B–100, apolipoprotein B–100; LDL-C, low-density lipoprotein cholesterol; mRNA, messenger ribonucleic acid; RNase H, ribonuclease H; VLDL, very low density lipoprotein. Reprinted from the Journal of the American College of Cardiology, Vol. 62/No.23, Thomas GS, Cromwell WC, Ali S, Chin W, Flaim JD, Davidson M, Mipomersen, an apolipoprotein B synthesis inhibitor, reduces atherogenic lipoproteins in patients with severe hypercholesterolemia at high cardiovascular risk: A Randomized, Double-Blind, Placebo-Controlled Trial, pp 2178–2184, Copyright 2013, with permission from the American College of Cardiology Foundation. Journal of Clinical Lipidology  , DOI: ( /j.jacl ) Copyright © 2016 Sanofi-Genzyme Terms and Conditions

3 Figure 2 Pediatric patient flow in randomized controlled trial and open-label extension. ALT, alanine aminotransferase; AST, aspartate aminotransferase; OLE, open-label extension study; RCT, randomized controlled trial. Journal of Clinical Lipidology  , DOI: ( /j.jacl ) Copyright © 2016 Sanofi-Genzyme Terms and Conditions

4 Figure 3 Low-density lipoprotein cholesterol concentration at measured time points in the randomized controlled trial and the open-label extension study. LDL-C, low-density lipoprotein cholesterol; OLE, open-label extension study; RCT, randomized controlled trial. Journal of Clinical Lipidology  , DOI: ( /j.jacl ) Copyright © 2016 Sanofi-Genzyme Terms and Conditions

5 Figure 4 Apo B concentration at measured time points in the randomized controlled trial and the open-label extension study. apo B, apolipoprotein B; OLE, open-label extension study; RCT, randomized controlled trial. Journal of Clinical Lipidology  , DOI: ( /j.jacl ) Copyright © 2016 Sanofi-Genzyme Terms and Conditions

6 Figure 5 Lipoprotein(a) concentration at measured time points in the randomized controlled trial and the open-label extension study. Lp(a), lipoprotein(a); OLE, open-label extension study; RCT, randomized controlled trial. Journal of Clinical Lipidology  , DOI: ( /j.jacl ) Copyright © 2016 Sanofi-Genzyme Terms and Conditions

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