Indication

Familial chylomicronemia syndrome

Familial chylomicronemia syndrome is a rare, autosomal recessive disorder of lipid metabolism primarily caused by biallelic loss-of-function variants in the lipoprotein lipase (*LPL*) gene or related cofactor genes. Read more about Familial chylomicronemia syndrome

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Common Familial chylomicronemia syndrome therapies.

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TRYNGOLZA (olezarsen sodium)

ANTIHYPERLIPIDEMIC - APOLIPOPROTEIN SYNTHESIS INHIBITORS·HYPERTRIGLYCERIDEMIA·+1

Ionis Pharmaceuticals, Inc.

REDEMPLO (plozasiran sodium)

ANTIHYPERLIPIDEMIC - APOLIPOPROTEIN SYNTHESIS INHIBITORS·FAMILIAL CHYLOMICRONEMIA SYNDROME

ARROWHEAD PHARM

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About Familial chylomicronemia syndrome.

Impaired lipolytic processing leads to marked accumulation of dietary chylomicrons in the circulation, resulting in severe and refractory hypertriglyceridemia, typically exceeding 880 to 1,000 mg/dL.

Affecting individuals across pediatric and adult populations, the condition predominantly impacts the digestive and vascular systems, predisposing patients to recurrent, potentially life-threatening acute pancreatitis.

Defining clinical features also include chronic or episodic abdominal pain, eruptive xanthomas, lipemia retinalis, and hepatosplenomegaly.

nih.gov
Familial chylomicronemia syndrome is a rare, autosomal recessive disorder of lipid metabolism primarily caused by biallelic loss-of-function variants in the lipoprotein lipase (*LPL*) gene or related cofactor genes Affecting individuals across pediatric and adult populations, the condition predominantly impacts the digestive and vascular systems, predisposing patients to recurrent, potentially life-threatening acute pancreatitis
lipid.org
Familial chylomicronemia syndrome is a rare, autosomal recessive disorder of lipid metabolism primarily caused by biallelic loss-of-function variants in the lipoprotein lipase (*LPL*) gene or related cofactor genes Impaired lipolytic processing leads to marked accumulation of dietary chylomicrons in the circulation, resulting in severe and refractory hypertriglyceridemia, typically exceeding 880 to 1,000 mg/dL
nih.gov
Impaired lipolytic processing leads to marked accumulation of dietary chylomicrons in the circulation, resulting in severe and refractory hypertriglyceridemia, typically exceeding 880 to 1,000 mg/dL Affecting individuals across pediatric and adult populations, the condition predominantly impacts the digestive and vascular systems, predisposing patients to recurrent, potentially life-threatening acute pancreatitis Defining clinical features also include chronic or episodic abdominal pain, eruptiv...
rarediseases.org
Impaired lipolytic processing leads to marked accumulation of dietary chylomicrons in the circulation, resulting in severe and refractory hypertriglyceridemia, typically exceeding 880 to 1,000 mg/dL
endocrine.org
Affecting individuals across pediatric and adult populations, the condition predominantly impacts the digestive and vascular systems, predisposing patients to recurrent, potentially life-threatening acute pancreatitis
nih.gov
Defining clinical features also include chronic or episodic abdominal pain, eruptive xanthomas, lipemia retinalis, and hepatosplenomegaly
medlineplus.gov
Defining clinical features also include chronic or episodic abdominal pain, eruptive xanthomas, lipemia retinalis, and hepatosplenomegaly

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