Antisense Inhibition of Apolipoprotein C-III in Patients with Hypertriglyceridemia.

  title={Antisense Inhibition of Apolipoprotein C-III in Patients with Hypertriglyceridemia.},
  author={Daniel Gaudet and Veronica J Alexander and Brenda F. Baker and Diane Brisson and Karine Tremblay and Walter Singleton and Richard S. Geary and Steven G. Hughes and Nicholas J. Viney and Mark J. Graham and Rosanne M. Crooke and Joseph L. Witztum and John D. Brunzell and John J P Kastelein},
  journal={The New England journal of medicine},
  volume={373 5},
BACKGROUND Apolipoprotein C-III (APOC3) is a key regulator of plasma triglyceride levels. Elevated triglyceride levels are associated with a risk of adverse cardiovascular events and pancreatitis. ISIS 304801 is a second-generation antisense inhibitor of APOC3 synthesis. METHODS We conducted a randomized, double-blind, placebo-controlled, dose-ranging, phase 2 study to evaluate ISIS 304801 in untreated patients with fasting triglyceride levels between 350 mg per deciliter (4.0 mmol per liter… 

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Results support the continued development of ISIS 304801 for the treatment of patients who remain at risk for cardiovascular events and pancreatitis because of very high triglyceride levels and further support the relationship between apoC‐III and triglycerides.

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