Reduction of elevated plasma globotriaosylsphingosine in patients with classic Fabry disease following enzyme replacement therapy

Fabry disease is treated by two-weekly infusions with α-galactosidase A, which is deficient in this X-linked globotriaosylceramide (Gb3) storage disorder. Elevated plasma globotriaosylsphingosine (lysoGb3) is a hallmark of classical Fabry disease. We investigated effects of enzyme replacement therap...

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Veröffentlicht in:	Biochimica et biophysica acta 2011-01, Vol.1812 (1), p.70-76
Hauptverfasser:	van Breemen, Mariëlle J., Rombach, Saskia M., Dekker, Nick, Poorthuis, Ben J., Linthorst, Gabor E., Zwinderman, Aeilko H., Breunig, Frank, Wanner, Christoph, Aerts, Johannes M., Hollak, Carla E.
Format:	Artikel
Sprache:	eng
Schlagworte:	Adolescent Adult Aged alpha-Galactosidase - immunology alpha-Galactosidase - therapeutic use Antibodies - blood Antibodies - immunology Child Child, Preschool Drug Administration Schedule Enzyme replacement therapy (ERT) Enzyme Replacement Therapy - methods Fabry disease Fabry Disease - blood Fabry Disease - drug therapy Female Globotriaosylsphingosine Glycolipids - blood Humans Isoenzymes - therapeutic use Lipids - blood LysoGb3 Male Middle Aged Sphingolipids - blood Treatment Outcome Young Adult
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