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RNA Interference Therapy Targeting Apolipoprotein C-III in Hypertriglyceridemia.

Daniel Gaudet, Peter Clifton, David Sullivan, John Baker, Christian Schwabe, Susan Thackwray, Russell Scott, James Hamilton, Bruce Given, Stacey Melquist, Rong Zhou, Ting Chang, Javier San Martin, Gerald F Watts, Ira J Goldberg, Joshua W Knowles, Robert A Hegele, Christie M Ballantyne
NEJM Evid 2023 December
APOC3-Targeting RNAi for HypertriglyceridemiaThis randomized controlled trial examined the safety and side effects of the small interfering RNA ARO-APOC3 in healthy volunteers and patients with hypertriglyceridemia and chylomicronemia. ARO-APOC3 was associated with few adverse events and no dose-limiting toxicities.

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