Abstract
Lysosomal acid lipase deficiency — an autosomal recessive storage disease that commonly manifests as elevated liver enzymes, dyslipidemia, hepatomegaly, and liver fibrosis — is an underappreciated cause of cirrhosis in both children and adults. In a manufacturer-sponsored, multicenter, phase III, double-blind trial, researchers evaluated the efficacy of sebelipase alfa enzyme replacement in patients aged ≥4 years with this disorder. Participants were randomized to …
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