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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Dades bibliogràfiques
Publicat a:Mol Genet Metab Rep
Autors principals: Beck, Michael, Hughes, Derralynn, Kampmann, Christoph, Larroque, Sylvain, Mehta, Atul, Pintos-Morell, Guillem, Ramaswami, Uma, West, Michael, Wijatyk, Anna, Giugliani, Roberto
Format: Artigo
Idioma:Inglês
Publicat: Elsevier 2015
Matèries:
SI:Therapy
Accés en línia: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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