Long-term outcomes with agalsidase alfa enzyme replacement therapy : analysis using deconstructed composite events
Beck, Michael (University Medical Center, Mainz, Germany)
Hughes, Derralynn A (University College London)
Kampmann, Christoph (University Medical Center, Mainz, Germany)
Pintos-Morell, Guillem (Institut Germans Trias i Pujol. Hospital Universitari Germans Trias i Pujol)
Ramaswami, Uma (University College London)
West, Michael L. (Dalhousie University, Halifax, Canada)
Giugliani, Roberto (Universidade Federal do Rio Grande do Sul, Brazil)
Universitat Autònoma de Barcelona

Date:	2017
Abstract:	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.
Note:	Altres ajuts: This work was supported by Shire International GmbH.
Rights:	Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, i la comunicació pública de l'obra, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original. No es permet la creació d'obres derivades.
Language:	Anglès
Document:	Article ; recerca ; Versió publicada
Subject:	Egfr, estimated glomerular filtration rate ; ERT, enzyme replacement therapy ; FOS, Fabry Outcome Survey ; LVH, left ventricular hypertrophy ; NE, not estimable ; Fabry disease ; Enzyme replacement therapy ; Agalsidase alfa ; Long-term effectiveness
Published in:	Molecular Genetics and Metabolism Reports, Vol. 14 (november 2017) , p. 31-35, ISSN 2214-4269

DOI: 10.1016/j.ymgmr.2017.10.008
PMID: 29159077

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Research literature > UAB research groups literature > Research Centres and Groups (research output) > Health sciences and biosciences > Institut d'Investigació en Ciencies de la Salut Germans Trias i Pujol (IGTP)
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Articles > Published articles