LAL-D: A Life-Threatening Genetic Disease with Progressive, Multiorgan Damage Leading To Premature Death

Lysosomal Acid Lipase Deficiency (LAL-D), historically known as Wolman disease and cholesteryl ester storage disease (CESD), may lead to severe systemic consequences including fibrosis, cirrhosis, cardiovascular complications, and malabsorption.1,2

LAL-D patients are deficient in the vital LAL enzyme, resulting in significant morbidities and may lead to premature death1


Percentage of patients who progressed to fibrosis, cirrhosis, or liver transplant within 3 years of first clinical manifestation.3


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