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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 that can lead to life-threatening liver or heart problems. The disease, affecting up to two people per million births, is commonly diagnosed during the first two-to-four months of infancy, the FDA said in a news release. Life expectancy depends on the disease's severity and its complications, the agency said. Kanuma is human Lal that's produced by genetically modified chickens in their egg whites. Neither the chicken or its eggs are allowed in the food supply, the FDA said. The injected drug is ... Read more

Related support groups: Sebelipase Alfa, Kanuma, Lysosomal Acid Lipase Deficiency

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 cells of various tissues that can lead to liver and cardiovascular disease and other complications. Wolman disease often presents during infancy (around 2 to 4 months of age) and is a rapidly progressive disease. Patients with Wolman disease rarely survive beyond the first year of life. CESD is a milder, later-onset form of LAL deficiency and presents in early childhood or later. Life expectancy of patients with CESD depends on the severity of the disease and associated complications. Wolman disease ... Read more

Related support groups: Sebelipase Alfa, Kanuma

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