FDA approves first drug to treat a rare enzyme disorder in pediatric and adult patients

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Title: FDA approves first drug to treat a rare enzyme disorder in pediatric and adult patients

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FDA approves first drug to treat a rare enzyme disorder in pediatric and adult patients

12/08/2015 10:43 AM EST

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.

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