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Cost-effectiveness of enzyme replacement therapy for Fabry disease

✍ Scribed by Saskia M Rombach, Carla EM Hollak, Gabor E Linthorst…

Book ID
120697088
Publisher
BioMed Central
Year
2013
Tongue
English
Weight
263 KB
Volume
8
Category
Article
ISSN
1750-1172

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## ABSTRACT Fabry disease is a multisystem disorder associated with wide variability in clinical expression. Fabry disease is an X‐linked lysosomal storage disorder caused by a deficiency of α‐galactosidase A. The enzyme defect leads to the systemic accumulation of glycosphingolipids with α‐galacto