The Medical Letter on Drugs and Therapeutics
Sebelipase Alfa (Kanuma) for Lysosomal Acid Lipase Deficiency (online only)
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The FDA has approved sebelipase alfa (Kanuma – Alexion), a recombinant form of lysosomal acid lipase (LAL), for enzyme replacement therapy in patients with LAL deficiency. Sebelipase alfa is the first drug to be approved in the US for treatment of LAL deficiency, a rare autosomal recessive storage disease.

LAL DEFICIENCY — LAL is a lysosomal glycoprotein enzyme that catalyzes the hydrolysis of cholesteryl esters and triglycerides. A complete deficiency of the enzyme (Wolman disease; estimated prevalence 1:500,000 live births) causes malabsorption, growth failure, hepatomegaly, adrenal cortical insufficiency, and death within the first year of life. Partial LAL deficiency (cholesteryl ester storage disease; estimated prevalence 1:40,000), which can present later in childhood or in adulthood, is associated with gastrointestinal symptoms, hepatosplenomegaly, elevated transaminase levels, and dyslipidemia, ... more      

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Title: Sebelipase Alfa (Kanuma) for Lysosomal Acid Lipase Deficiency (online only)
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