Internal medicine

Endocrinology

Adipose tissue

Enzyme

Pancreatitis

Acute pancreatitis

Cholesterol

Genetic disorder

Triglyceride

Disease

Lipase

Biochemistry

Apolipoprotein B

Medicine

Biology

Lipoprotein lipase

New England Journal of Medicine

Familial chylomicronemia syndrome is a rare genetic disorder that is caused by loss of lipoprotein lipase activity and characterized by chylomicronemia and recurrent episodes of pancreatitis. There are no effective therapies. In an open-label study of three patients with this syndrome, antisense-mediated inhibition of hepatic APOC3 mRNA with volanesorsen led to decreased plasma apolipoprotein C-III and triglyceride levels.

Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome