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

BK, Burton; M, Balwani; F, Feillet; I, Barić; TA, Burrow; Grande C, Camarena; M, Coker; A, Consuelo-Sánchez; P, Deegan; Rocco M, Di; GM, Enns; R, Erbe; F, Ezgu; C, Ficicioglu; KN, Furuya; J, Kane; C, Laukaitis; E, Mengel; EG, Neilan; S, Nightingale; H, Peters; M, Scarpa; KO, Schwab; V, Smolka; V, Valayannopoulos; M, Wood; Z, Goodman; Y, Yang; S, Eckert; S, Rojas-Caro; AG, Quinn

doi:10.1056/nejmoa1501365

Public

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

Shared by NobleBlocks on Sep 1, 2015 • 12:00 AM UTC

Authors:

Burton BK

Balwani M

Feillet F

Abstract

Background Lysosomal acid lipase is an essential lipid-metabolizing enzyme that breaks down endocytosed lipid particles and regulates lipid metabolism. We conducted a phase 3 trial of enzyme-replacement therapy in children and adults with lysosomal acid lipase deficiency, an underappreciated cause o...

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