Journal article
Changes in plasma and urine globotriaosylceramide levels do not predict Fabry disease progression over 1 year of agalsidase alfa
R Schiffmann, M Ries, D Blankenship, K Nicholls, A Mehta, JTR Clarke, RD Steiner, M Beck, BA Barshop, W Rhead, M West, R Martin, D Amato, N Nair, P Huertas
Genetics in Medicine | Published : 2013
DOI: 10.1038/gim.2013.56
Abstract
Purpose:Globotriaosylceramide concentrations were assessed as potential predictors of change from baseline after 12 months by estimated glomerular filtration rate and left-ventricular mass index using pooled data from three randomized, placebo-controlled agalsidase alfa trials and open-label extensions of patients with Fabry disease.Methods:Males (aged 18 years or older) with Fabry disease received agalsidase alfa (0.2 mg/kg every other week for 12 months). A backward-elimination approach evaluated potential predictors (baseline estimated glomerular filtration rate and left-ventricular mass index; age at first dose; baseline and change from baseline at 12 months of globotriaosylceramide (uri..
View full abstractGrants
Funding Acknowledgements
These studies and analyses were funded by Shire Human Genetic Therapies (HGT). Medical writing support for this article was provided by Ray Beck of UBC-Envision Group and was funded by Shire HGT.R.S. has received honoraria and research support from Shire Human Genetic Therapies (HGT), Amicus Therapeutics, and Genzyme. K.N. has served on the Fabry Outcomes Survey advisory board that is funded by Shire HGT and has received travel and research support from Shire HGT, Genzyme, and Amicus; her center participates in clinical trials funded by Shire HGT and Amicus. A. M. has received research support and speaking honoraria from Shire HGT, Genzyme, and Actelion. J.T.R.C. has received research funding, consultancy fees, and/or speaker fees from Shire HGT, Genzyme, and Actelion. R. D. S. has participated in clinical trials funded by TKT/Shire HGT, Actelion, Genzyme, Protalix/Pfizer, and BioMarin. He has had research support from TKT and has received speaking or consulting fees from TKT/Shire HGT, Amicus, Actelion, Genzyme, Zacharon, and BioMarin. M. B. has received unrestricted grants, honoraria, and travel support from Shire HGT, Genzyme, BioMarin, and Actelion. B. A. B. has received research support from Genzyme and has served on an advisory board for BioMarin. W. R. has received research funding, consultancy fees, and/or speaker fees from Shire HGT, Genzyme, Hyperion, Ucyclyd, and Actelion and has served on advisory boards for Shire HGT. M. W. has received research support from Shire HGT and Genzyme; has received speaking honoraria from Shire HGT, Genzyme, Amicus Therapeutics, and Sumitomo Pharma; and has participated in advisory boards for Shire HGT. M. R. was an employee of Shire HGT from 2006 to 2009; he has served on advisory boards for Alexion, GSK, and Amicus and has received consultancy honoraria from Alexion, Oxyrane, and Shire HGT. P. H. was an employee of Shire HGT from 2008 to 2011. R. M. and N. N are employees of Shire HGT. D. A. was an employee of Shire HGT from 2010 to 2012. D. B. declares no potential competing interests.