Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome

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

doi:10.1056/nejmoa1715944

Public

Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome

Published in New England Journal of Medicine • Aug 7, 2019

Authors:

Joseph L. Witztum

Daniel Gaudet

Steven D. Freedman

Abstract

BACKGROUND: mRNA with volanesorsen led to decreased plasma apolipoprotein C-III and triglyceride levels. METHODS: We conducted a phase 3, double-blind, randomized 52-week trial to evaluate the safety and effectiveness of volanesorsen in 66 patients with familial chylomicronemia syndrome. Patients we...

Subject

Medicine

Triglyceride

Internal medicine

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