Long-term effectiveness of agalsidase alfa enzyme replacement in Fabry disease: A Fabry Outcome Survey analysis

Outcomes from 5 years of treatment with agalsidase alfa enzyme replacement therapy (ERT) for Fabry disease in patients enrolled in the Fabry Outcome Survey (FOS) were compared with published findings for untreated patients with Fabry disease. Data were extracted from FOS, a Shire-sponsored database,...

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Detalhes bibliográficos
Publicado no:Mol Genet Metab Rep
Main Authors: Beck, Michael, Hughes, Derralynn, Kampmann, Christoph, Larroque, Sylvain, Mehta, Atul, Pintos-Morell, Guillem, Ramaswami, Uma, West, Michael, Wijatyk, Anna, Giugliani, Roberto
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2015
Assuntos:
SI:Therapy
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4750577/
https://ncbi.nlm.nih.gov/pubmed/26937390
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2015.02.002
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