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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Detalles Bibliográficos
Autores principales: Beck, Michael, Hughes, Derralynn, Kampmann, Christoph, Pintos-Morell, Guillem, Ramaswami, Uma, West, Michael L., Giugliani, Roberto
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2017
Materias:
Short Communication
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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author Beck, Michael
Hughes, Derralynn
Kampmann, Christoph
Pintos-Morell, Guillem
Ramaswami, Uma
West, Michael L.
Giugliani, Roberto
author_facet Beck, Michael
Hughes, Derralynn
Kampmann, Christoph
Pintos-Morell, Guillem
Ramaswami, Uma
West, Michael L.
Giugliani, Roberto
author_sort Beck, Michael
collection PubMed
description 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 cerebrovascular or renal events, cerebrovascular events were more frequent in females than males, and males were more likely to experience cardiac and renal events at a younger age than females.
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spelling pubmed-56845032017-11-20 Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events Beck, Michael Hughes, Derralynn Kampmann, Christoph Pintos-Morell, Guillem Ramaswami, Uma West, Michael L. Giugliani, Roberto Mol Genet Metab Rep Short Communication 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 cerebrovascular or renal events, cerebrovascular events were more frequent in females than males, and males were more likely to experience cardiac and renal events at a younger age than females. Elsevier 2017-11-09 /pmc/articles/PMC5684503/ /pubmed/29159077 http://dx.doi.org/10.1016/j.ymgmr.2017.10.008 Text en © 2017 The Authors http://creativecommons.org/licenses/by-nc-nd/4.0/ This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).
spellingShingle Short Communication
Beck, Michael
Hughes, Derralynn
Kampmann, Christoph
Pintos-Morell, Guillem
Ramaswami, Uma
West, Michael L.
Giugliani, Roberto
Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events
title Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events
title_full Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events
title_fullStr Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events
title_full_unstemmed Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events
title_short Long-term outcomes with agalsidase alfa enzyme replacement therapy: Analysis using deconstructed composite events
title_sort long-term outcomes with agalsidase alfa enzyme replacement therapy: analysis using deconstructed composite events
topic Short Communication
url 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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