Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study.

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作者： Eric L,Wallace ^[1] ; Ozlem,Goker-Alpan ^[2] ; William R,Wilcox ^[3] ; Myrl,Holida ^[4] ; John,Bernat ^[4] ; Nicola,Longo ^[5] ; Aleš,Linhart ^[6] ; Derralynn A,Hughes ^[7] ; Robert J,Hopkin ^[8] ; Camilla,Tøndel ^[9] ; Mirjam,Langeveld ^[10] ; Pilar,Giraldo ^[11] ; Antonio,Pisani ^[12] ; Dominique Paul,Germain ^[13] ; Ankit,Mehta ^[14] ; Patrick B,Deegan ^[15] ; Maria Judit,Molnar ^[16] ; Damara,Ortiz ^[17] ; Ana,Jovanovic ^[18] ; Michael,Muriello ^[19] ; Bruce A,Barshop ^[20] ; Virginia,Kimonis ^[21] ; Bojan,Vujkovac ^[22] ; Albina,Nowak ^[23] ; Tarekegn,Geberhiwot ^[24] ; Ilkka,Kantola ^[25] ; Jasmine,Knoll ^[26] ; Stephen,Waldek ^[27] ; Khan,Nedd ^[28] ; Amel,Karaa ^[29] ; Einat,Brill-Almon ^[30] ; Sari,Alon ^[31] ; Raul,Chertkoff ^[30] ; Rossana,Rocco ^[32] ; Anat,Sakov ^[33] ; David G,Warnock ^[34]

作者单位： Department of Medicine, Division of Nephrology, The University of Alabama at Birmingham, Birmingham, Alabama, USA. ^[1] Lysosomal and Rare Disorders Research and Treatment Center, Inc, Fairfax, Virginia, USA. ^[2] Department of Human Genetics, Emory University School of Medicine, Atlanta, Georgia, USA. ^[3] Department of Pediatrics, University of Iowa Hospitals and Clinics, Iowa City, Iowa, USA. ^[4] Department of Pediatrics, Division of Medical Genetics, University of Utah Health, Salt Lake City, Utah, USA. ^[5] Department of Internal Medicine, School of Medicine, Charles University and General University Hospital, Prague, Czech Republic. ^[6] Lysosomal Storage Disorders Unit, Royal Free London NHS Foundation Trust and University College London, London, UK. ^[7] Department of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA. ^[8] Department of Clinical Science, University of Bergen, Bergen, Norway.;Department of Pediatrics, Haukeland University Hospital, Bergen, Norway. ^[9] Academisch Medisch Centrum Universiteit van Amsterdam, Amsterdam, The Netherlands. ^[10] Unidad de Investigación Traslacional. Hospital Universitario Miguel Servet, Instituto de Investigación Sanitaria Aragón (IIS Aragón), Zaragoza, Spain.;Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER-ISCIII), Zaragoza, Spain. ^[11] Department of Public Health, Universita degli Studi di Napoli Federico II, Napoli, Italy. ^[12] Division of Medical Genetics, University of Versailles, Garches, France. ^[13] Baylor University Medical Center at Dallas, Dallas, Texas, USA. ^[14] Lysosmal Disorders Unit, Department of Medicine, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK. ^[15] Institute of Genomic Medicine and Rare Disorders, Semmelweis University Clinical Center, Budapest, Hungary. ^[16] Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, USA. ^[17] Mark Holland Metabolic Unit, Northern Care Alliance NHS Foundation Trust, Greater Manchester, UK. ^[18] Department of Pediatrics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA. ^[19] Department of Pediatrics, University of California San Diego, La Jolla, California, USA. ^[20] Department of Pediatrics, University of California Irvine, Irvine, California, USA. ^[21] Department of Internal Medicine, General Hospital Slovenj Gradec, Slovenj Gradec, Slovenia. ^[22] Department of Endocrinology and Clinical Nutrition, University Hospital Zurich and University of Zurich, Zurich, Switzerland. ^[23] Department of Diabetes, Endocrinology and Metabolism, Queen Elizabeth Hospital Birmingham, Birmingham, UK. ^[24] Division of Medicine, Turku University Hospital, Turku, Finland. ^[25] Phoenix Children's Hospital, Phoenix, Arizona, USA. ^[26] University of Sunderland, Sunderland, UK. ^[27] Infusion Associates, Grand Rapids, Michigan, USA. ^[28] Massachusetts General Hospital for Children, Boston, Massachusetts, USA. ^[29] Protalix Biotherapeutics, Carmiel, Israel. ^[30] Product Development, Protalix Biotherapeutics, Carmiel, Israel. ^[31] Chiesi Farmaceutici SpA, Parma, Italy. ^[32] DataSights, Haifa, Israel. ^[33] Department of Medicine, Division of Nephrology, The University of Alabama at Birmingham, Birmingham, Alabama, USA dwarnock@uab.edu. ^[34]