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eRAM

encyclopedia of Rare Disease Annotation for Precision Medicine




Disease lysosomal acid lipase deficiency
Comorbidity C0023890|cirrhosis
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PubMedID- 26352813 We conducted a phase 3 trial of enzyme-replacement therapy in children and adults with lysosomal acid lipase deficiency, an underappreciated cause of cirrhosis and severe dyslipidemia.

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