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Kanuma News

Kanuma Approved for Rare Missing-Enzyme Disease

Posted 12 Dec 2015 by

TUESDAY, Dec. 8, 2015 – Kanuma (sebelipase alfa) has been approved by the U.S. Food and Drug Administration as the first treatment for a rare but often-deadly disease caused by an enzyme deficiency. People with Lal deficiency, also known as Wolman disease or cholesteryl ester storage disease, produce little or none of the enzyme lysosomal acid lipase (Lal). This causes fat buildup in the body ...

FDA Approves Kanuma (sebelipase alfa) for Lysosomal Acid Lipase Deficiency

Posted 10 Dec 2015 by

December 8, 2015 – Today, the U.S. Food and Drug Administration approved Kanuma (sebelipase alfa) as the first treatment for patients with a rare disease known as lysosomal acid lipase (LAL) deficiency. Patients with LAL deficiency (also known as Wolman disease and cholesteryl ester storage disease [CESD]) have no or little LAL enzyme activity. This results in a build-up of fats within the ...

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Lysosomal Acid Lipase Deficiency

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