A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency
Volume: 373, Issue: 11, Pages: 1010 - 1020
Published: Sep 10, 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...
Paper Details
Title
A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency
Published Date
Sep 10, 2015
Volume
373
Issue
11
Pages
1010 - 1020
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