Familial Lecithin:Cholesterol Acyltransferase Deficiency: First-in-Human Treatment with Enzyme Replacement

BACKGROUND: Humans with familial lecithin:cholesterol acyltransferase (LCAT) deficiency (FLD) have extremely low or undetectable HDL-C levels and by early adulthood develop many manifestations of the disorder, including corneal opacities, anemia, and renal disease. OBJECTIVE: To determine if infusio...

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Gepubliceerd in:J Clin Lipidol
Hoofdauteurs: Shamburek, Robert D., Bakker-Arkema, Rebecca, Auerbach, Bruce J., Krause, Brian R., Homan, Reynold, Amar, Marcelo J., Freeman, Lita A., Remaley, Alan T.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2015
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Article
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4826469/
https://ncbi.nlm.nih.gov/pubmed/27055967
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.jacl.2015.12.007
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