Original paper
Sebelipase Alfa: A Review in Lysosomal Acid Lipase Deficiency
Volume: 16, Issue: 6, Pages: 461 - 468
Published: Nov 23, 2016
Abstract
Sebelipase alfa (Kanuma®, Kanuma™), the first commercially available recombinant human lysosomal acid lipase (LAL), is approved in various countries worldwide, including those of the EU, the USA and Japan, as a long-term enzyme replacement therapy for patients diagnosed with LAL deficiency (LAL-D), an ultra-rare, autosomal recessive, progressive metabolic liver disease. In an ongoing study in nine infants presenting with early-onset LAL-D...
Paper Details
Title
Sebelipase Alfa: A Review in Lysosomal Acid Lipase Deficiency
Published Date
Nov 23, 2016
Volume
16
Issue
6
Pages
461 - 468
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Notes
History