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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)
  • Abstract:

    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

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Last update 4 October 2019

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