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Lysosomal Acid Lipase Deficiency News
Related terms: Wolman's Disease
Posted 6 Feb 2017 by Drugs.com
MONDAY, Feb. 6, 2017 – A set of screening tests designed to detect four rare metabolic disorders in newborns has been approved by the U.S. Food and Drug Administration. The "Seeker" system of diagnostics is designed to screen for Mucopolysaccharidosis Type 1, Pompe, Gaucher and Fabry. These are the first tests approved to screen for the inherited disorders, which affect proteins that normally ...
Posted 12 Dec 2015 by Drugs.com
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 ...