A phase III, open-label clinical trial evaluating pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease previously treated with other enzyme replacement therapies.

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作者： Myrl,Holida ^[1] ; Aleš,Linhart ^[2] ; Antonio,Pisani ^[3] ; Nicola,Longo ^[4] ; François,Eyskens ^[5] ; Ozlem,Goker-Alpan ^[6] ; Eric,Wallace ^[7] ; Patrick,Deegan ^[8] ; Camilla,Tøndel ^[9] ; Ulla,Feldt-Rasmussen ^[10] ; Derralynn,Hughes ^[11] ; Anat,Sakov ^[12] ; Rossana,Rocco ^[13] ; Einat Brill,Almon ^[14] ; Sari,Alon ^[14] ; Raul,Chertkoff ^[14] ; David G,Warnock ^[7] ; Stephen,Waldek ^[15] ; William R,Wilcox ^[16] ; John A,Bernat ^[1]

作者单位： Division of Medical Genetics and Genomics, Stead Family Department of Pediatrics, University of Iowa, Iowa City, Iowa, USA. ^[1] Charles University, General University Hospital, Prague, Czech Republic. ^[2] Department of Public Health, University Federico II of Naples, Naples, Italy. ^[3] Pediatrics Medical Genetics, University of Utah, Salt Lake City, Utah, USA. ^[4] Antwerp University Hospital UZA, Edegem, Belgium. ^[5] Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, Virginia, USA. ^[6] University of Alabama at Birmingham, Birmingham, Alabama, USA. ^[7] Lysosomal Disorders Unit, Cambridge University Hospitals NHS Foundation Trust and University of Cambridge, Cambridge, UK. ^[8] University of Bergen and Haukeland University Hospital, Bergen, Norway. ^[9] Department of Endocrinology and Metabolism, Rigshospitalet and Faculty of Health and Clinical Sciences, Copenhagen University, Copenhagen, Denmark. ^[10] LSDU, Royal Free London NHS Foundation Trust, and University College London, London, UK. ^[11] DataSights Ltd, Haifa, Israel. ^[12] Chiesi Farmaceutici S.p.A, Parma, Italy. ^[13] Department of Product Development, Protalix Biotherapeutics, Carmiel, Israel. ^[14] University of Sunderland, Sunderland, UK. ^[15] Department of Human Genetics, Emory University School of Medicine, Atlanta, Georgia, USA. ^[16]

关键词 Fabry disease eGFR enzyme replacement therapy lysosomal storage disorders lyso‐Gb3 pegunigalsidase alfa