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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...
Autores principales: | Beck, Michael, Hughes, Derralynn, Kampmann, Christoph, Pintos-Morell, Guillem, Ramaswami, Uma, West, Michael L., Giugliani, Roberto |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2017
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5684503/ https://www.ncbi.nlm.nih.gov/pubmed/29159077 http://dx.doi.org/10.1016/j.ymgmr.2017.10.008 |
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