Consequences of involuntary dose reductions during shortage of agalsidase beta - lessons learned from patients suffering from Fabry disease
- At: 2014 FIP Congress in Bangkok (Thailand)
- Type: Presentation
- By: LINTHORST, Gabor (Academic Medical Center, Endocrinology and metabolism, Amsterdam, Netherlands)
- Co-author(s): Smid, Bouwien (Academic Medical Center, Amsterdam, Netherlands)
Hollak, Carla (Academic Medical Center, Amsterdam, Netherlands)
Background: Treatment with biweekly infusions with alpha-Galactosidase A (aGal A) is the only specific treatment for Fabry disease (aGal A deficiency), using either agalsidase alfa 0,2mg/kg/2wks or agalsidase beta 1,0 mg/kg/2wks (authorized in the EU)). In 2009 due to a viral contamination in the agalsidase beta production facility, a worldwide.. The access to the whole abstract and the presentation file is available to FIP members and to congress participants of that specific congress.