Sebelipase alfa improves atherogenic biomarkers in adults and children with lysosomal acid lipase deficiency.
Journal of Clinical Lipidology（2018）
Abstract
•Sebelipase alfa treatment resulted in improvement in most atherogenic biomarkers.•The improvements were sustained with long-term sebelipase alfa therapy.•Sebelipase alfa was well tolerated.
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Key words
Lysosomal acid lipase deficiency,Sebelipase alfa,Dyslipidemia,Enzyme replacement therapy,Wolman disease,Cholesteryl ester storage disease
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