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Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain

BACKGROUND: Fabry disease is an X-linked lysosomal storage disorder caused by α-galactosidase A deficiency leading to renal, cardiac, cerebrovascular disease and premature death. Treatment with α-galactosidase A (enzyme replacement therapy, ERT) stabilises disease in some patients, but long term eff...

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Detalles Bibliográficos
Autores principales:	Rombach, Saskia M, Smid, Bouwien E, Bouwman, Machtelt G, Linthorst, Gabor E, Dijkgraaf, Marcel G W, Hollak, Carla E M
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2013
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3626869/ https://www.ncbi.nlm.nih.gov/pubmed/23531228 http://dx.doi.org/10.1186/1750-1172-8-47

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