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Four-Year Prospective Clinical Trial of Agalsidase Alfa in Children with Fabry Disease |
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| Authors: | Raphael Schiffmann Rick A. Martin Tyler Reimschisel Karen Johnson Victoria Castaneda Y. Howard Lien Gregory M. Pastores Christoph Kampmann Markus Ries Joe T.R. Clarke |
| Affiliation: | a Institute of Metabolic Disease, Baylor Research Institute, Dallas, TX b Developmental and Metabolic Neurology Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD c Washington University School of Medicine, St Louis, MO d University of Tennessee Health Science Center, Memphis, TN e East Tennessee Children's Hospital, Knoxville, TN f Arizona Health Sciences Center, Tucson, AZ g New York University School of Medicine, New York, NY h University Children's Hospital, Mainz, Germany i Hospital for Sick Children, Toronto, Ontario, Canada |
| Abstract: | |
| Keywords: | BPI, Brief Pain Index eGFR, Estimated glomerular filtration rate Gb3, Globotriaosylceramide LVM, Left ventricular mass |
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