Expert opinion on temporary treatment recommendations for Fabry disease during the shortage of enzyme replacement therapy (ERT)

Mol Genet Metab. 2011 Jan;102(1):99-102. doi: 10.1016/j.ymgme.2010.11.155. Epub 2010 Nov 30.


The shortage of enzyme for treatment of Fabry disease has caused anxiety among patients, physicians and governments. Following a request from the European Medicines Agency, consensus was reached on the temporary prioritization of patients for treatment based on disease severity and potential reversibility. Advice on follow-up of patients was agreed upon. This consensus is proposed to support the temporary guidelines issued throughout the period of ERT shortage, which will most likely last until April 2011.

Publication types

  • Consensus Development Conference
  • Practice Guideline

MeSH terms

  • Enzyme Replacement Therapy
  • Humans
  • Isoenzymes / administration & dosage
  • Isoenzymes / supply & distribution*
  • alpha-Galactosidase / administration & dosage
  • alpha-Galactosidase / supply & distribution*


  • Isoenzymes
  • alpha-Galactosidase
  • agalsidase beta