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

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  • Additional Information
    • Contributors:
      Burton, Barbara K; Balwani, Manisha; Feillet, Françoi; 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.
    • Publication Date:
      2015
    • Collection:
      Padua Research Archive (IRIS - Università degli Studi di Padova)
    • 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.
    • Relation:
      info:eu-repo/semantics/altIdentifier/pmid/26352813; info:eu-repo/semantics/altIdentifier/wos/WOS:000360959000006; volume:373; issue:11; firstpage:1010; lastpage:1020; numberofpages:11; journal:NEW ENGLAND JOURNAL OF MEDICINE; http://hdl.handle.net/11577/3168673; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-84941312383
    • Accession Number:
      10.1056/NEJMoa1501365
    • Online Access:
      http://hdl.handle.net/11577/3168673
      https://doi.org/10.1056/NEJMoa1501365
    • Rights:
      info:eu-repo/semantics/openAccess
    • Accession Number:
      edsbas.507E0C9A