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Effectiveness of agalsidase alfa enzyme replacement in Fabry disease: cardiac outcomes after 10 years’ treatment

BACKGROUND: To explore long-term effects of agalsidase alfa on Fabry disease cardiomyopathy in adults. METHODS: Retrospective analysis of prospectively collected data at a single center in Mainz, Germany, revealed that 45 adult patients (21 men, 24 women) had received agalsidase alfa for approximate...

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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Kampmann, Christoph, Perrin, Amandine, Beck, Michael
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2015
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4587871/
https://ncbi.nlm.nih.gov/pubmed/26416388
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-015-0338-2
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