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Related to Acid lipase deficiency: Lipoprotein lipase deficiency
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Lysosomal Acid Lipase Deficiency is a rare, autosomal recessive lysosomal storage disorder (LSD) that is caused by a marked decrease in LAL enzyme activity.
Each year, Synageva joins efforts with The Global Genes Project, a leading non-profit organization for rare and genetic diseases, in a volunteer fundraiser with proceeds to benefit the Support Organization for Lysosomal Acid Lipase Deficiency Advocacy, Care and Expertise (LAL Solace) organization and other LAL Deficiency patient organizations and patient advocacy groups.
Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for Kanuma(TM) (sebelipase alfa) for the treatment of lysosomal acid lipase deficiency (LAL Deficiency).
Lysosomal Acid Lipase Deficiency is a rare, autosomal recessive LSD that is caused by a marked decrease in LAL enzyme activity.
EP2613798 B1 relating to the treatment of lysosomal acid lipase deficiency (LAL Deficiency).