Sebelipase alfa in children and adults with lysosomal acid lipase deficiency: Final results of the ARISE study

•Long-term sebelipase alfa treatment provided multiple benefits for patients with LAL-D.•Improvements in liver and lipid parameters with sebelipase alfa were sustained.•Liver histology was mostly stable or improved from baseline with sebelipase alfa.•Improvements were noted in all age groups and irrespective of lipid medication use.•Sebelipase alfa therapy for up to 5 years was generally well tolerated.