Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events

This is a retrospective analysis of Fabry Outcome Survey data from children/adults (n = 677) receiving agalsidase alfa enzyme replacement therapy for a median of 3 years, examining cerebrovascular, cardiac, and renal morbidity endpoints separately. Cardiac events occurred at younger ages than cerebr...

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Bibliographic Details
Published in:Mol Genet Metab Rep
Main Authors: Beck, Michael, Hughes, Derralynn, Kampmann, Christoph, Pintos-Morell, Guillem, Ramaswami, Uma, West, Michael L., Giugliani, Roberto
Format: Artigo
Language:Inglês
Published: Elsevier 2017
Subjects:
Short Communication
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC5684503/
https://ncbi.nlm.nih.gov/pubmed/29159077
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2017.10.008
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