Lysosomal acid lipase is an essential lipid-metabolizing enzyme that breaks down endocytosed lipid particles and regulates lipid metabolism. We conducted a phase 3 trial of enzyme-replacement therapy in children and adults with lysosomal acid lipase deficiency, an underappreciated cause of cirrhosis and severe dyslipidemia.

A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency

SCARPA, MAURIZIO;
2015

Abstract

Lysosomal acid lipase is an essential lipid-metabolizing enzyme that breaks down endocytosed lipid particles and regulates lipid metabolism. We conducted a phase 3 trial of enzyme-replacement therapy in children and adults with lysosomal acid lipase deficiency, an underappreciated cause of cirrhosis and severe dyslipidemia.
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11577/3168673
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