Novel treatment options for lysosomal acid lipase deficiency: critical appraisal of sebelipase alfa

Kim Su,1 Emma Donaldson,1 Reena Sharma2 1Division of Gastroenterology/Hepatology, 2The Mark Holland Metabolic Unit, Salford Royal Hospital NHS Foundation Trust, Salford, UK Abstract: Lysosomal acid lipase deficiency (LAL-D) is a rare disorder of cholesterol metabolism with an autosomal recessive mod...

Full description

Bibliographic Details
Main Authors: Su K, Donaldson E, Sharma R
Format: Article
Language:English
Published: Dove Medical Press 2016-10-01
Series:The Application of Clinical Genetics
Subjects:
Online Access:https://www.dovepress.com/novel-treatment-options-for-lysosomal-acid-lipase-deficiency-critical--peer-reviewed-article-TACG