Alipogene Tiparvovec: A Review of Its Use in Adults with Familial Lipoprotein Lipase Deficiency

Alipogene tiparvovec (Glybera ® ; AMT-011, AAV1-LPL S447X ) is an adeno-associated virus serotype 1-based gene therapy for adult patients with familial lipoprotein lipase (LPL) deficiency (LPLD) and suffering from severe or multiple pancreatitis attacks despite dietary fat restrictions. It is admini...

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Veröffentlicht in:	Drugs (New York, N.Y.) N.Y.), 2015-02, Vol.75 (2), p.175-182
1. Verfasser:	Scott, Lesley J.
Format:	Artikel
Sprache:	eng
Schlagworte:	Adis Drug Evaluation Adult Adults Dependovirus - genetics Enzymes Gene therapy Genetic testing Genetic Therapy - methods Genetic Vectors - adverse effects Genetic Vectors - genetics Genomes Humans Hyperlipoproteinemia Type I - genetics Hyperlipoproteinemia Type I - therapy Internal Medicine Lipoproteins Medicine Medicine & Public Health Metabolism Mitochondrial DNA Musculoskeletal system Mutation Oils & fats Pancreatitis Pharmacology/Toxicology Pharmacotherapy Ribosomal DNA Vectors (Biology)
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