Ace the Case: A 28-Year-Old Man With Hepatomegaly and Dyslipidemia


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Question 1
Which of the following best describes the mechanism of action for sebelipase alpha?
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Correct answer: D.

Rationale: Sebelipase alpha is a recombinant lysosomal lipase, FDA approved in December 2015 to treat lysosomal acid lipase deficiency in infants and adults.11 It is administered once a week via intravenous infusion in people with rapidly progressing disease in the first 6 months of life, and every other week with less aggressive disease.11