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Novel Therapeutics for Familial Chylomicronemia Syndrome

21 Apr 2025

Abstract excerpt

PURPOSE OF REVIEW: This review discusses new treatment approaches for familial chylomicronemia syndrome (FCS), a rare disorder affecting triglyceride metabolism. The focus is on antisense oligonucleotides (ASO) and small-interfering RNA (siRNA) therapies targeting APOC3 and angiopoietin-like protein 3 (ANGPTL3). RECENT FINDINGS: Volanesorsen, an ASO targeting APOC3, has shown effectiveness in managing FCS,...

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PMID: 40257687
DOI: 10.1007/s11883-025-01295-x

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