Migalastat HCl Reduces Globotriaosylsphingosine (Lyso-Gb(3)) in Fabry Transgenic Mice and in the Plasma of Fabry Patients
Brandy Young-Gqamana, Nastry Brignol, Hui-Hwa Chang, Richie Khanna, Rebecca Soska, Maria Fuller, Sheela A Sitaraman, Dominique P Germain, Roberto Giugliani, Derralynn A Hughes, Atul Mehta, Kathy Nicholls, Pol Boudes, David J Lockhart, Kenneth J Valenzano, Elfrida R Benjamin
PLoS One | PUBLIC LIBRARY SCIENCE | Published : 2013
Brandy Young-Gqamana is a former employee of Amicus Therapeutics. Nastry Brignol, Hui-Hwa Chang, Richie Khanna, Rebecca Soska, Sheela A. Sitaraman, Pol Boudes, David J. Lockhart, Kenneth J. Valenzano, and Elfrida R. Benjamin are employed by Amicus Therapeutics and are shareholders in the company. Maria Fuller was a collaborator with Amicus Therapeutics. All other authors were investigators on the Phase 2 clinical studies of migalastat HCl. Dominique P. Germain has received research funding, consultancy fees, and travel expenses from Genzyme and Shire HGT. Roberto Giugliani is a consultant and investigator for Actelion, Amicus, BioMarin, Genzyme and Shire HGT. Derralynn A. Hughes is a consultant for Amicus, Shire HGT, Genzyme, Actelion, has been on a Speaker's Bureau for Amicus, Shire HGT, Genzyme, and Actelion, and has received grants from Amicus, Shire HGT, and Genzyme. Atul Mehta has received honoraria, research funding, consultancy fees, and travel expenses from Shire HGT, Genzyme, Actelion, Protalix, and Amicus. Kathy Nicholls has received travel and research support and speaker's honoraria from Amicus, Shire HGT and Genzyme. Amicus Therapeutics funded the research and any publication fees. The alpha-Gal A (Fabrazyme) used in this study is a product produced by Genzyme. Maria Fuller is employed by SA Pathology, Adelaide. There are no further patents, products in development or marketed products to declare. This does not alter the authors' adherence to all of the PLOS ONE policies on sharing data and materials.