Clinical considerations for the treatment of patients with familial chylomicronemia syndrome using a hepatic-targeted <i>APOC3</i> antisense oligonucleotide.

ABSTRACT: Familial chylomicronemia syndrome (FCS) is a rare, typically debilitating genetic disorder of extreme hypertriglyceridemia associated with high triglyceride levels and elevated risk for recurrent acute pancreatitis. Diagnosis of FCS is frequently delayed due to its rarity, and treatment options are limited. Patients often report history of acute pancreatitis or associated symptoms, including chronic or recurrent abdominal pain, weakness, and fatigue. The hallmark of chylomicronemia (extreme hypertriglyceridemia) syndromes, including FCS, is extremely high triglyceride levels ≥880 mg/dL (10 mmol/L) resistant to conventional triglyceride-lowering medications including statins, fibrates, and omega-3 fatty acids. Validated clinical scoring tools or genetic testing can support diagnosis. Patien

SUBMITTER: Bajaj A 

PROVIDER: S-EPMC12686656 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature