Effects of enzyme replacement therapy with agalsidase alfa on glomerular filtration rate in patients with Fabry disease: preliminary data

Acta Paediatr Suppl. 2003 Dec;92(443):14-5; discussion 5. doi: 10.1111/j.1651-2227.2003.tb00214.x.


Progressive deposition of globotriaosylceramide results in severe complications involving the kidney, heart and brain in both hemizygous male and heterozygous female patients with Fabry disease. Analysis of renal data from FOS--the Fabry Outcome Survey--suggests that enzyme replacement therapy with agalsidase alfa can significantly improve renal function in patients with Fabry disease, at least in those with a mild decrease in glomerular filtration rate, and may also be able to slow down the natural decline in renal function in patients with a moderate reduction in glomerular filtration rate.

Conclusion: Initial results from the large cohort of patients within FOS indicate that treatment with agalsidase alfa has beneficial effects on kidney function in patients with Fabry disease.

MeSH terms

  • Fabry Disease / drug therapy*
  • Fabry Disease / physiopathology*
  • Glomerular Filtration Rate / drug effects*
  • Humans
  • Isoenzymes / pharmacology
  • Isoenzymes / therapeutic use*
  • Recombinant Proteins
  • alpha-Galactosidase / pharmacology
  • alpha-Galactosidase / therapeutic use*


  • Isoenzymes
  • Recombinant Proteins
  • agalsidase alfa
  • alpha-Galactosidase