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Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome

by Arca, Marcello , Blom, Dirk J , Williams, Karren R , Geary, Richard S , Soran, Handrean , Freedman, Steven D , Alexander, Veronica J , Civeira, Fernando , Digenio, Andres , Tsimikas, Sotirios , O’Dea, Louis , Gaudet, Daniel , Hughes, Steven G , Hemphill, Linda , Witztum, Joseph L , Stroes, Erik S.G , Bruckert, Eric , Bergeron, Jean , Yang, Qingqing

in Adult / Aged / Analysis of Variance / Antisense therapy / Apolipoprotein C-III / Apolipoprotein C-III - antagonists & inhibitors / Apolipoprotein C-III - blood / Apolipoprotein C-III - genetics / Apolipoproteins / Clinical trials / Double-Blind Method / Drug dosages / Fasting / Fatty acids / Female / Genetic disorders / Humans / Hyperlipoproteinemia Type I - blood / Hyperlipoproteinemia Type I - drug therapy / Injection / Injections, Subcutaneous - adverse effects / Laboratories / Life Sciences / Lipase / Lipoprotein lipase / Lipoproteins / Male / Middle Aged / mRNA / Mutation / Oligonucleotides - administration & dosage / Oligonucleotides - adverse effects / Oligonucleotides - therapeutic use / Pancreatitis / Pharmaceuticals / Platelet Count / Platelets / RNA, Messenger - antagonists & inhibitors / Statistical analysis / Thrombocytopenia / Thrombocytopenia - chemically induced / Triglycerides - blood / Young Adult

2019

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Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome

2019

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Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome

2019

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Journal Article

Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome

Arca, Marcello,

Blom, Dirk J,

Williams, Karren R,

Geary, Richard S,

Soran, Handrean,

Freedman, Steven D,

Alexander, Veronica J,

Civeira, Fernando,

Digenio, Andres,

Tsimikas, Sotirios,

O’Dea, Louis,

Gaudet, Daniel,

Hughes, Steven G,

Hemphill, Linda,

Witztum, Joseph L,

Stroes, Erik S.G,

Bruckert, Eric,

Bergeron, Jean,

Yang, Qingqing

2019

Overview

This phase 3 trial showed that treatment with volanesorsen, an antisense oligonucleotide drug complementary to mRNA encoding apolipoprotein C-III, resulted in a mean reduction in triglyceride levels of 77% over the course of 3 months.

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Publisher

Massachusetts Medical Society

Subject

Adult

/ Aged

/ Analysis of Variance

/ Antisense therapy

/ Apolipoprotein C-III

/ Apolipoprotein C-III - antagonists & inhibitors

/ Apolipoprotein C-III - blood