Article
Alipogene tiparvovec for the treatment of lipoprotein lipase deficiency.
Drugs of today (Barcelona, Spain : 1998) - 1 Mar 2013
Haddley K
Abstract excerpt
Alipogene tiparvovec is the first adeno-associated virus (AAV)-mediated gene therapy to be approved for the treatment of a metabolic disorder. Lipoprotein lipase (LPL) deficiency (LPLD) is a rare autosomal-recessive disorder in which gene mutations cause the production of a catalytically inactive enzyme required for plasma triglyceride hydrolysis. The resultant hypertriglyceridemia causes frequent abdominal pain,...
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