학술논문
A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency.
Document Type
Article
Author
Source
Subject
*THERAPEUTIC use of enzymes
*LYSOSOMAL storage diseases
*THERAPEUTICS
*CIRRHOSIS of the liver
*DYSLIPIDEMIA
*INBORN errors of metabolism
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Language
ISSN
0028-4793
Abstract
The article discusses a phase three clinical trial of enzyme-replacement therapy in patients with lysosomal acid lipase deficiency. The deficiency is said to be an underappreciated cause of cirrhosis and severe dyslipidemia. The trial involved 66 children and adults administered with enzyme-replacement therapy with sebelipase alfa to evaluate its safety and effectiveness. The study found that sebelipase alfa therapy reduced multiple disease-related hepatic and lipid abnormalities.