Volanesorsen and triglyceride levels in familial chylomicronemia syndrome

New England Journal of Medicine Aug 13, 2019

Witztum JL, Gaudet D, Freedman SD, et al. - Researchers evaluated the safety and efficacy of volanesorsen in a phase 3, double-blind, randomized, 52-week trial. The study sample consisted of 66 patients with familial chylomicronemia syndrome who were randomized in a 1:1 ratio to receive volanesorsen or placebo. Volanesorsen treatment reduced triglyceride levels to < 750 mg per deciliter in 77% of patients with familial chylomicronemia syndrome. Adverse events commonly encountered included thrombocytopenia and injection-site reactions.

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