Sebelipase alfa

Review
In: LiverTox: Clinical and Research Information on Drug-Induced Liver Injury [Internet]. Bethesda (MD): National Institute of Diabetes and Digestive and Kidney Diseases; 2012.
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Excerpt

Lysosomal acid lipase deficiency is an inherited condition which underlies Wolman disease and cholesteryl ester storage disease. The current standard treatment for these two conditions is enzyme replacement therapy using infusions of recombinant forms of lysosomal acid lipase. Enzyme replacement therapy is generally well tolerated and has not been linked to serum enzyme elevations or to instances of clinically apparent acute liver injury.

Publication types

  • Review