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

  • Burton, Barbara K
  • Balwani, Manisha
  • Feillet, François
  • Barić, Ivo
  • Burrow, T Andrew
  • Camarena Grande, Carmen
  • Coker, Mahmut
  • Consuelo-Sánchez, Alejandra
  • Deegan, Patrick
  • Di Rocco, Maja
  • Enns, Gregory M
  • Erbe, Richard
  • Ezgu, Fatih
  • Ficicioglu, Can
  • Furuya, Katryn N
  • Kane, John
  • Laukaitis, Christina
  • Mengel, Eugen
  • Neilan, Edward G
  • Nightingale, Scott
  • Peters, Heidi
  • Scarpa, Maurizio
  • Schwab, K Otfried
  • Smolka, Vratislav
  • Valayannopoulos, Vassili
  • Wood, Marnie
  • Goodman, Zachary
  • Yang, Yijun
  • Eckert, Stephen
  • Rojas-Caro, Sandra
  • Quinn, Anthony G
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Publication date
September 2015
Icon DOI
10.1056/NEJMoa1501365
Publisher
Massachusetts Medical Society
Journal
New England Journal of Medicine

Abstract

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 of cirrhosis and severe dyslipidemia

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