A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency

Abstract

Sebelipase alfa therapy resulted in a reduction in multiple disease-related hepatic and lipid abnormalities in children and adults with lysosomal acid lipase deficiency. (Funded by Synageva BioPharma and others; ARISE ClinicalTrials.gov number, NCT01757184.).