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Antisense and
Apo B Synthesis Inhibition
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Content
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Apo B-100: Long-recognized as a Promising Target
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Antisense Technology Provided a Method for Targeting Apo B
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Antisense Drug Discovery Selectively Targets
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Mipomersen – A 2nd Generation Antisense Drug
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Mipomersen Crosses the Hepatocyte and Nuclear Membrane
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Mechanism of ActionRNase H-mediated Degradation of Target mRNA
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Metabolism of MipomersenDrug Metabolism and Disposition
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Targeting Apo B Production Reduces Formation of VLDL
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The Development of Mipomersen
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Reductions in Total Cholesterol Correlate With Apo B mRNA in
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Pharmacology Proof of ConceptApoB Inhibition
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Phase 2: Hypercholesterolemia, Monotherapy
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Mipomersen Significantly Reduced LDL-C in Statin-Intolerant Patients
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Mipomersen Pharmacokineticand Pharmacodynamic Properties
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MipomersenSummary of Phase 3 Clinical Studies
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Phase 3 Extension Study
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Phase 3 Extension StudyStudy Design
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Phase 3 Extension StudySustained Reduction of Atherogenic
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Phase 3 Extension StudySafety Data
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Phase 3 Extension StudySafety Summary
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Phase 3 Long Term Extension Study Overall Summary
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Emerging Therapies in Development
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