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Retrospective study of long-term outcomes of enzyme replacement therapy in Fabry disease: Analysis of prognostic factors.

Despite enzyme replacement therapy, disease progression is observed in patients with Fabry disease. Identification of factors that predict disease progression is needed to refine guidelines on initiation and cessation of enzyme replacement therapy. To study the association of potential biochemical a...

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Principais autores: Maarten Arends, Marieke Biegstraaten, Derralynn A Hughes, Atul Mehta, Perry M Elliott, Daniel Oder, Oliver T Watkinson, Frédéric M Vaz, André B P van Kuilenburg, Christoph Wanner, Carla E M Hollak
Format: Artigo
Sprog:Inglês
Udgivet: Public Library of Science (PLoS) 2017-01-01
Serier:PLoS ONE
Online adgang:https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0182379&type=printable
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