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August 7, 2019New England Journal of Medicine682 citationsOpen Access

Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome

JWJoseph L. WitztumDGDaniel GaudetSFSteven D. Freedman

Structured PICO

Does volanesorsen reduce triglyceride levels in patients with familial chylomicronemia syndrome?

P
Population
Patients with familial chylomicronemia syndrome
I
Intervention
Volanesorsen
O
Outcome
Triglyceride levelssurrogate

Volanesorsen effectively lowers triglyceride levels in patients with familial chylomicronemia syndrome, though it is associated with thrombocytopenia and injection-site reactions.

Abstract

Volanesorsen lowered triglyceride levels to less than 750 mg per deciliter in 77% of patients with familial chylomicronemia syndrome. Thrombocytopenia and injection-site reactions were common adverse events. (Funded by Ionis Pharmaceuticals and Akcea Therapeutics; APPROACH Clinical Trials.gov number, NCT02211209.).

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Cite This Study

Witztum et al. (2019) studied this question.

synapsesocial.com/papers/69dd5f2a656f15f1fc1013c7https://doi.org/10.1056/nejmoa1715944
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