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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.
Wallace, Eric L; Goker-Alpan, Ozlem; Wilcox, William R; Holida, Myrl; Bernat, John; Longo, Nicola; Linhart, Ales; Hughes, Derralynn A; Hopkin, Robert J; Tøndel, Camilla; Langeveld, Mirjam; Giraldo, Pilar; Pisani, Antonio; Germain, Dominique Paul; Mehta, Ankit; Deegan, Patrick B; Molnar, Maria Judit; Ortiz, Damara; Jovanovic, Ana; Muriello, Michael; Barshop, Bruce A; Kimonis, Virginia; Vujkovac, Bojan; Nowak, Albina; Geberhiwot, Tarekegn; Kantola, Ilkka; Knoll, Jasmine; Waldek, Stephen; Nedd, Khan; Karaa, Amel; Brill-Almon, Einat; Alon, Sari; Chertkoff, Raul; Rocco, Rossana; Sakov, Anat; Warnock, David G.
Afiliación
  • Wallace EL; Department of Medicine, Division of Nephrology, The University of Alabama at Birmingham, Birmingham, Alabama, USA.
  • Goker-Alpan O; Lysosomal and Rare Disorders Research and Treatment Center, Inc, Fairfax, Virginia, USA.
  • Wilcox WR; Department of Human Genetics, Emory University School of Medicine, Atlanta, Georgia, USA.
  • Holida M; Department of Pediatrics, University of Iowa Hospitals and Clinics, Iowa City, Iowa, USA.
  • Bernat J; Department of Pediatrics, University of Iowa Hospitals and Clinics, Iowa City, Iowa, USA.
  • Longo N; Department of Pediatrics, Division of Medical Genetics, University of Utah Health, Salt Lake City, Utah, USA.
  • Linhart A; Department of Internal Medicine, School of Medicine, Charles University and General University Hospital, Prague, Czech Republic.
  • Hughes DA; Lysosomal Storage Disorders Unit, Royal Free London NHS Foundation Trust and University College London, London, UK.
  • Hopkin RJ; Department of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
  • Tøndel C; Department of Clinical Science, University of Bergen, Bergen, Norway.
  • Langeveld M; Department of Pediatrics, Haukeland University Hospital, Bergen, Norway.
  • Giraldo P; Academisch Medisch Centrum Universiteit van Amsterdam, Amsterdam, The Netherlands.
  • Pisani A; Unidad de Investigación Traslacional. Hospital Universitario Miguel Servet, Instituto de Investigación Sanitaria Aragón (IIS Aragón), Zaragoza, Spain.
  • Germain DP; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER-ISCIII), Zaragoza, Spain.
  • Mehta A; Department of Public Health, Universita degli Studi di Napoli Federico II, Napoli, Italy.
  • Deegan PB; Division of Medical Genetics, University of Versailles, Garches, France.
  • Molnar MJ; Baylor University Medical Center at Dallas, Dallas, Texas, USA.
  • Ortiz D; Lysosmal Disorders Unit, Department of Medicine, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK.
  • Jovanovic A; Institute of Genomic Medicine and Rare Disorders, Semmelweis University Clinical Center, Budapest, Hungary.
  • Muriello M; Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, USA.
  • Barshop BA; Mark Holland Metabolic Unit, Northern Care Alliance NHS Foundation Trust, Greater Manchester, UK.
  • Kimonis V; Department of Pediatrics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.
  • Vujkovac B; Department of Pediatrics, University of California San Diego, La Jolla, California, USA.
  • Nowak A; Department of Pediatrics, University of California Irvine, Irvine, California, USA.
  • Geberhiwot T; Department of Internal Medicine, General Hospital Slovenj Gradec, Slovenj Gradec, Slovenia.
  • Kantola I; Department of Endocrinology and Clinical Nutrition, University Hospital Zurich and University of Zurich, Zurich, Switzerland.
  • Knoll J; Department of Diabetes, Endocrinology and Metabolism, Queen Elizabeth Hospital Birmingham, Birmingham, UK.
  • Waldek S; Division of Medicine, Turku University Hospital, Turku, Finland.
  • Nedd K; Phoenix Children's Hospital, Phoenix, Arizona, USA.
  • Karaa A; University of Sunderland, Sunderland, UK.
  • Brill-Almon E; Infusion Associates, Grand Rapids, Michigan, USA.
  • Alon S; Massachusetts General Hospital for Children, Boston, Massachusetts, USA.
  • Chertkoff R; Protalix Biotherapeutics, Carmiel, Israel.
  • Rocco R; Product Development, Protalix Biotherapeutics, Carmiel, Israel.
  • Sakov A; Protalix Biotherapeutics, Carmiel, Israel.
  • Warnock DG; Chiesi Farmaceutici SpA, Parma, Italy.
J Med Genet ; 2023 Nov 08.
Article en En | MEDLINE | ID: mdl-37940383
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Idioma: En Revista: J Med Genet Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Idioma: En Revista: J Med Genet Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos