Sebelipase alfa over 52 weeks reduces serum transaminases, liver volume and improves serum lipids in patients with lysosomal acid lipase deficiency

BACKGROUND AND AIMS: Lysosomal Acid Lipase Deficiency is an autosomal recessive enzyme deficiency resulting in lysosomal accumulation of cholesteryl esters and triglycerides. LAL-CL04, an ongoing extension study, investigates the long-term effects of sebelipase alfa, a recombinant human lysosomal ac...

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Autors principals: Valayannopoulos, Vassili, Malinova, Vera, Honzík, Tomas, Balwani, Manisha, Breen, Catherine, Deegan, Patrick B., Enns, Gregory M., Jones, Simon A., Kane, John P., Stock, Eveline O., Tripuraneni, Radhika, Eckert, Stephen, Schneider, Eugene, Hamilton, Gavin, Middleton, Michael S., Sirlin, Claude, Kessler, Bruce, Bourdon, Christopher, Boyadjiev, Simeon A., Sharma, Reena, Twelves, Chris, Whitley, Chester B., Quinn, Anthony G.
Format: Artigo
Idioma:Inglês
Publicat: 2014
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4203712/
https://ncbi.nlm.nih.gov/pubmed/24993530
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.jhep.2014.06.022
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