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Long-term outcome of enzyme-replacement therapy in advanced Fabry disease: evidence for disease progression towards serious complications

OBJECTIVE: The long-term effects of enzyme-replacement therapy (ERT) in Fabry disease are unknown. Thus, the aim of this study was to determine whether ERT in patients with advanced Fabry disease affects progression towards ‘hard’ clinical end-points in comparison with the natural course of the dise...

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Detalles Bibliográficos
Autores principales: Weidemann, F, Niemann, M, Störk, S, Breunig, F, Beer, M, Sommer, C, Herrmann, S, Ertl, G, Wanner, C
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BlackWell Publishing Ltd 2013
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4282332/
https://www.ncbi.nlm.nih.gov/pubmed/23586858
http://dx.doi.org/10.1111/joim.12077