Safety and efficacy of enzyme replacement therapy in the nephropathy of Fabry disease

Fernando C Fervenza1, Roser Torra2, David G Warnock31Division of Nephrology and Hypertension, Mayo Clinic College of Medicine, Rochester, MN, USA; 2Department of Nephrology, Fundaci&oacute; Puigvert, Barcelona, Spain; 3Division of Nephrology, University of Alabama at Birmingham, Birmingham,...

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Autores principales:	Fernando C Fervenza, Roser Torra, David G Warnock
Formato:	article
Lenguaje:	EN
Publicado:	Dove Medical Press 2008
Materias:	Medicine (General) R5-920
Acceso en línea:	https://doaj.org/article/9f552c5ce0f44462b60e7169845b7b6f
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spelling	oai:doaj.org-article:9f552c5ce0f44462b60e7169845b7b6f2021-12-02T07:34:38ZSafety and efficacy of enzyme replacement therapy in the nephropathy of Fabry disease1177-54751177-5491https://doaj.org/article/9f552c5ce0f44462b60e7169845b7b6f2008-11-01T00:00:00Zhttp://www.dovepress.com/safety-and-efficacy-of-enzyme-replacement-therapy-in-the-nephropathy-o-a2573https://doaj.org/toc/1177-5475https://doaj.org/toc/1177-5491Fernando C Fervenza1, Roser Torra2, David G Warnock31Division of Nephrology and Hypertension, Mayo Clinic College of Medicine, Rochester, MN, USA; 2Department of Nephrology, Fundaci&oacute; Puigvert, Barcelona, Spain; 3Division of Nephrology, University of Alabama at Birmingham, Birmingham, AL, USAAbstract: 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.Keywords: agalsidase, enzyme replacement therapy, Fabry nephropathy, anti-proteinuric therapy Fernando C FervenzaRoser TorraDavid G WarnockDove Medical PressarticleMedicine (General)R5-920ENBiologics: Targets & Therapy, Vol 2008, Iss Issue 4, Pp 823-843 (2008)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine (General) R5-920
spellingShingle	Medicine (General) R5-920 Fernando C Fervenza Roser Torra David G Warnock Safety and efficacy of enzyme replacement therapy in the nephropathy of Fabry disease
description	Fernando C Fervenza1, Roser Torra2, David G Warnock31Division of Nephrology and Hypertension, Mayo Clinic College of Medicine, Rochester, MN, USA; 2Department of Nephrology, Fundaci&oacute; Puigvert, Barcelona, Spain; 3Division of Nephrology, University of Alabama at Birmingham, Birmingham, AL, USAAbstract: 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.Keywords: agalsidase, enzyme replacement therapy, Fabry nephropathy, anti-proteinuric therapy
format	article
author	Fernando C Fervenza Roser Torra David G Warnock
author_facet	Fernando C Fervenza Roser Torra David G Warnock
author_sort	Fernando C Fervenza
title	Safety and efficacy of enzyme replacement therapy in the nephropathy of Fabry disease
title_short	Safety and efficacy of enzyme replacement therapy in the nephropathy of Fabry disease
title_full	Safety and efficacy of enzyme replacement therapy in the nephropathy of Fabry disease
title_fullStr	Safety and efficacy of enzyme replacement therapy in the nephropathy of Fabry disease
title_full_unstemmed	Safety and efficacy of enzyme replacement therapy in the nephropathy of Fabry disease
title_sort	safety and efficacy of enzyme replacement therapy in the nephropathy of fabry disease
publisher	Dove Medical Press
publishDate	2008
url	https://doaj.org/article/9f552c5ce0f44462b60e7169845b7b6f
work_keys_str_mv	AT fernandocfervenza safetyandefficacyofenzymereplacementtherapyinthenephropathyoffabrydisease AT rosertorra safetyandefficacyofenzymereplacementtherapyinthenephropathyoffabrydisease AT davidgwarnock safetyandefficacyofenzymereplacementtherapyinthenephropathyoffabrydisease
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Safety and efficacy of enzyme replacement therapy in the nephropathy of Fabry disease

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