Zimran A, Brill-Almon E, Chertkoff R, et al. Pivotal trial with plant cell–expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease. Blood. 2011;118(22):5767–5773

Autor: Gheona Altarescu, Atul Mehta, Gloria Duran, Milan Petakov, Glen Park, Hanna Rosenbaum, Francisco Blanco-Favela, Sergio Solorio-Meza, Mici Phillips, Deborah Elstein, Rene Heitner, David Aviezer, Dominick Amato, Mali Szleifer, Fiorina Giona, Sharon Hashmueli, Einat Brill-Almon, Ari Zimran, Raul Chertkoff, Eduardo Terreros Muñoz, Pilar Giraldo

On page 5767 in the 24 November 2011 issue, there are errors in the affiliations. The affiliation of the eleventh author (Heitner) is misspelled. The correct affiliation is University of Witwatersrand. The affiliation of the last author (Aviezer) is

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b3a45100e91f23fc64ce960e00214cc7
https://europepmc.org/articles/PMC3362370/

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.

Autor: 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.; Department of Pediatrics, Haukeland University Hospital, Bergen, Norway., Langeveld M; Academisch Medisch Centrum Universiteit van Amsterdam, Amsterdam, The Netherlands., Giraldo P; 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., Pisani A; Department of Public Health, Universita degli Studi di Napoli Federico II, Napoli, Italy., Germain DP; Division of Medical Genetics, University of Versailles, Garches, France., Mehta A; Baylor University Medical Center at Dallas, Dallas, Texas, USA., Deegan PB; Lysosmal Disorders Unit, Department of Medicine, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK., Molnar MJ; Institute of Genomic Medicine and Rare Disorders, Semmelweis University Clinical Center, Budapest, Hungary., Ortiz D; Department of Pediatrics, UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, USA., Jovanovic A; Mark Holland Metabolic Unit, Northern Care Alliance NHS Foundation Trust, Greater Manchester, UK., Muriello M; Department of Pediatrics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA., Barshop BA; Department of Pediatrics, University of California San Diego, La Jolla, California, USA., Kimonis V; Department of Pediatrics, University of California Irvine, Irvine, California, USA., Vujkovac B; Department of Internal Medicine, General Hospital Slovenj Gradec, Slovenj Gradec, Slovenia., Nowak A; Department of Endocrinology and Clinical Nutrition, University Hospital Zurich and University of Zurich, Zurich, Switzerland., Geberhiwot T; Department of Diabetes, Endocrinology and Metabolism, Queen Elizabeth Hospital Birmingham, Birmingham, UK., Kantola I; Division of Medicine, Turku University Hospital, Turku, Finland., Knoll J; Phoenix Children's Hospital, Phoenix, Arizona, USA., Waldek S; University of Sunderland, Sunderland, UK., Nedd K; Infusion Associates, Grand Rapids, Michigan, USA., Karaa A; Massachusetts General Hospital for Children, Boston, Massachusetts, USA., Brill-Almon E; Protalix Biotherapeutics, Carmiel, Israel., Alon S; Product Development, Protalix Biotherapeutics, Carmiel, Israel., Chertkoff R; Protalix Biotherapeutics, Carmiel, Israel., Rocco R; Chiesi Farmaceutici SpA, Parma, Italy., Sakov A; DataSights, Haifa, Israel., Warnock DG; Department of Medicine, Division of Nephrology, The University of Alabama at Birmingham, Birmingham, Alabama, USA dwarnock@uab.edu.

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Taliglucerase alfa leads to favorable bone marrow responses in patients with type I Gaucher disease

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Inflammation, Oxidative Stress, and Endothelial Dysfunction in the Pathogenesis of Vascular Damage: Unraveling Novel Cardiovascular Risk Factors in Fabry Disease.

Autor: Faro, Denise Cristiana¹ (AUTHOR) denisefaro88@gmail.com, Di Pino, Francesco Lorenzo¹ (AUTHOR), Monte, Ines Paola¹ (AUTHOR) ines.monte@unict.it

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WS01.2 Phase II clinical trial results of alidornase alfa for the treatment of cystic fibrosis

Autor: Kerem, E., Blau, H., Shteinberg, M., Efrati, O., Alon, S., Dekel, E., Amit, B.C., Raul, C., Brill-Almon, E., Fux, L., Shaaltiel, Y., Bentur, L.

Publikováno v: In Journal of Cystic Fibrosis June 2017 16 Supplement 1:S1-S1