ArticleBiologics : targets & therapy2008
Safety and efficacy of enzyme replacement therapy in the nephropathy of Fabry disease.
Article in Biologics : targets & therapy, 2008. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
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Who cites it
15 citing papers in PubMed, 39 citations in OpenAlex.
- Targeted nanoliposomes to improve enzyme replacement therapy of Fabry disease.Science advances · 2024Article
- Management of Hypertension in Fabry Disease.Electrolyte & blood pressure : E & BP · 2023Review
- Agalsidase beta treatment slows estimated glomerular filtration rate loss in classic Fabry disease patients: results from an individual patient data meta-analysis.Clinical kidney journal · 2021Article
- Fabry disease: dose matters.Journal of the American Society of Nephrology : JASN · 2014Article
- Agalsidase benefits renal histology in young patients with Fabry disease.Journal of the American Society of Nephrology : JASN · 2013Article
- Development of a highly sensitive immuno-PCR assay for the measurement of α-galactosidase A protein levels in serum and plasma.PloS one · 2013Article
- Efficient uptake of recombinant α-galactosidase A produced with a gene-manipulated yeast by Fabry mice kidneys.Molecular medicine (Cambridge, Mass.) · 2012Article
- Mannose 6-phosphate receptor and sortilin mediated endocytosis of α-galactosidase A in kidney endothelial cells.PloS one · 2012Article
- Cardiomyopathy and response to enzyme replacement therapy in a male mouse model for Fabry disease.PloS one · 2012Article
- Receptor-mediated endocytosis of α-galactosidase A in human podocytes in Fabry disease.PloS one · 2011Article
- Scoring system for renal pathology in Fabry disease: report of the International Study Group of Fabry Nephropathy (ISGFN).Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association · 2010Article
- Characterization of Fabry mice treated with recombinant adeno-associated virus 2/8-mediated gene transfer.Journal of biomedical science · 2010Article
- Assessment of renal pathology and dysfunction in children with Fabry disease.Clinical journal of the American Society of Nephrology : CJASN · 2010Review
- Recommendations for the diagnosis and management of Fabry disease in pediatric patients: a document from the Rare Diseases Committee of the Brazilian Society of Nephrology (Comdora-SBN).Jornal brasileiro de nefrologiaReview
- Prevalence of Kidney and Urinary Tract Complications in Fabry Disease from 2000 to 2020: A Global Cohort Study Including 10,637 Patients.In vivo (Athens, Greece)Article
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Authors and funding
3 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Kidney involvement with progressive loss of kidney function (Fabry nephropathy) is an important complication of Fabry disease, an X-linked lysosomal storage disorder arising from deficiency of alpha-galactosidase activity. Clinical trials have shown that enzyme replacement therapy (ERT) with recombinant human alpha-galactosidase clears globotriaosylceramide from kidney cells, and can stabilize kidney function in patients with mild to moderate Fabry nephropathy. Recent trials show that patients with more advanced Fabry nephropathy and overt proteinuria do not respond as well to ERT alone, but can benefit from anti-proteinuric therapy given in conjunction with ERT. This review focuses on the use of enzyme replacement therapy with agalsidase-alfa and agalsidase-beta in adults with Fabry nephropathy. The current results are reviewed and evaluated. The issues of dosing of enzyme replacement therapy, the use of adjunctive agents to control urinary protein excretion, and the individual factors that affect disease severity are reviewed.
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