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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Autori principali:	Michael Beck, Derralynn Hughes, Christoph Kampmann, Sylvain Larroque, Atul Mehta, Guillem Pintos-Morell, Uma Ramaswami, Michael West, Anna Wijatyk, Roberto Giugliani
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	Elsevier 2015-06-01
Serie:	Molecular Genetics and Metabolism Reports
Soggetti:	Fabry disease Enzyme replacement therapy Agalsidase alfa Long-term effectiveness
Accesso online:	http://www.sciencedirect.com/science/article/pii/S2214426915000099
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