RT Journal Article
SR Electronic
A1 Vinall, Maria
T1 Sebelipase Alfa Normalizes ALT and Multiple other Disease-Related Abnormalities in Patients with LAL-D
JF MD Conference Express
YR 2014
FD SAGE Publications
VO 14
IS 48
SP 14
OP 15
DO 10.1177/155989771448008
UL http://mdc.sagepub.com/content/14/48/14.abstract
AB Lysosomal acid lipase deficiency (LAL-D) is an autosomal genetic recessive disorder that leads to an inability to break down lipid particles in the lysosome, and it is associated with early-onset cirrhosis and cardiovascular disease. This article discusses the Acid Lipase Replacement Investigating Safety and Efficacy trial [ARISE; NCT01757184] was a randomized, double-blind, placebo-controlled phase 3 study designed to evaluate sebelipase alfa in patients with LAL-D.