6533b858fe1ef96bd12b6324

RESEARCH PRODUCT

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

Roberta RicciAtul MehtaUfosi WidmerMathias BeckF DehoutAndreas Schwarting

subject

medicine.medical_specialtyGlobotriaosylceramideUrologyRenal functionchemistry.chemical_compoundInternal medicinemedicineHumansIn patientBeneficial effectsKidneybusiness.industryGeneral MedicineEnzyme replacement therapymedicine.diseaseFabry diseaseRecombinant ProteinsIsoenzymesEndocrinologymedicine.anatomical_structurechemistryalpha-GalactosidasePediatrics Perinatology and Child HealthFabry DiseasebusinessAgalsidase alfaGlomerular Filtration Rate

description

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.

yearjournalcountryeditionlanguage
2007-01-02Acta Paediatrica
https://doi.org/10.1111/j.1651-2227.2003.tb00214.x