Erik S G Stroes, Veronica J Alexander, Ewa Karwatowska-Prokopczuk, Robert A Hegele, Marcello Arca, Christie M Ballantyne, Handrean Soran, Thomas A Prohaska, Shuting Xia, Henry N Ginsberg, Joseph L Witztum, Sotirios Tsimikas
BACKGROUND: Familial chylomicronemia syndrome is a genetic disorder associated with severe hypertriglyceridemia and severe acute pancreatitis. Olezarsen reduces the plasma triglyceride level by reducing hepatic synthesis of apolipoprotein C-III. METHODS: In a phase 3, double-blind, placebo-controlled trial, we randomly assigned patients with genetically identified familial chylomicronemia syndrome to receive olezarsen at a dose of 80 mg or 50 mg or placebo subcutaneously every 4 weeks for 53 weeks...
April 7, 2024: New England Journal of Medicine