Genetics in Medicine Open (Jan 2023)
P005: Head-to-head trial of pegunigalsidase alfa vs agalsidase beta in Fabry disease: Phase 3 randomized, double-blind, BALANCE Study 2-year results
- John Bernat,
- Eric Wallace,
- Ozlem Goker-Alpan,
- William Wilcox,
- Myrl Holida,
- Nicola Longo,
- Derralynn Hughes,
- Pilar Giraldo,
- Maria Molnar,
- Damara Ortiz,
- Robert Hopkin,
- Camilla Tondel,
- Ales Linhart,
- Patrick Deegan,
- Ana Jovanovic,
- Michael Muriello,
- Bruce Barshop,
- Virginia Kimonis,
- Bojan Vujkovac,
- Albina Nowak,
- Tarekegn Hiwot,
- Antonio Pisani,
- Dominique Germain,
- Ilkka Kantola,
- Jasmine Knoll,
- Ankit Mehta,
- Stephen Waldek,
- Einat Almon,
- Sari Alon,
- Raul Chertkoff,
- Rossana Rocco,
- David Warnock
Affiliations
- John Bernat
- University of Iowa Hospitals and Clinics, Iowa City, IA
- Eric Wallace
- The University of Alabama at Birmingham, Birmingham, AL
- Ozlem Goker-Alpan
- Lysosomal and Rare Disorders Research and Treatment Center, Inc., Fairfax, VA
- William Wilcox
- Department of Human Genetics, Emory University, Atlanta, GA
- Myrl Holida
- University of Iowa Hospitals and Clinics, Iowa City, IA
- Nicola Longo
- University of Utah, Center for Clinical Translational Sciences, Salt Lake City, UT
- Derralynn Hughes
- Lysosomal Storage Disorders Unit, Royal Free London NHS Foundation Trust and University College London, London, UK
- Pilar Giraldo
- Quironsalud Hospital, Zaragoza, Spain
- Maria Molnar
- Institute of Genomic Medicine and Rare Disorders, Semmelweis University, Budapest, HUN
- Damara Ortiz
- UPMC Children’s Hospital of Pittsburgh, Pittsburgh, PA
- Robert Hopkin
- Cincinnati Children’s Hospital Medical Center, Cincinnati, OH
- Camilla Tondel
- Haukeland University Hospital, Renal Research Group, Bergen, NOR
- Ales Linhart
- Vseobecna fakultni nemocnice v Praze, Praha, CZE
- Patrick Deegan
- Addenbrooke’s Hospital, Cambridge University Hospitals, Cambridge, UK
- Ana Jovanovic
- Mark Holland Metabolic Unit, Northern Care Alliance NHS Foundation Trust, Greater Manchester, UK
- Michael Muriello
- Department of Pediatrics – Genetics Curative Network Building, Milwaukee, WI
- Bruce Barshop
- University of California San Diego, La Jolla, CA
- Virginia Kimonis
- University of California, Irvine institute for Clinical and Translational Science, CA
- Bojan Vujkovac
- Slovenj Gradec General Hospital, Slovenj Gradec, SI
- Albina Nowak
- University Hospital of Psychiatry Zürich, Department of internal medicine, CHE
- Tarekegn Hiwot
- Queen Elizabeth Hospital Birmingham, Edgbaston, Birmingham West Midlands, UK
- Antonio Pisani
- Azienda Ospedaliera Universitaria ''Federico II'', Dipartimento di Sanita Pubblica, Napoli, ITA
- Dominique Germain
- University of Versailles, Division of Medical Genetics, Garches, FRA
- Ilkka Kantola
- Department of Medicine, Turku University Hospital, Turku, FIN
- Jasmine Knoll
- Phoenix Children’s Hospital, Phoenix, AZ
- Ankit Mehta
- Baylor University Medical Center, Dallas, TX
- Stephen Waldek
- University of Sunderland, Sunderland, UK
- Einat Almon
- Protalix Biotherapeutics, Carmiel, ISL
- Sari Alon
- Protalix Biotherapeutics, Carmiel, ISL
- Raul Chertkoff
- Protalix Biotherapeutics, Carmiel, ISL
- Rossana Rocco
- Chiesi Farmaceutici S.p.A., Parma, ITA
- David Warnock
- The University of Alabama at Birmingham, Birmingham, AL
- Journal volume & issue
-
Vol. 1,
no. 1
p. 100015