We acknowledge the participation of study patients and their families and the expert assistance of all study-site coordinators and personnel. We also acknowledge the key contributions of our colleagues Dr. Ann Lowe and Ms. Mary Newman, as well as the many other BioMarin employees and consultants who performed important roles during the studies. Dr. Helen Nicely of BioMarin contributed to the editing of this document. This study was sponsored by BioMarin Pharmaceutical Inc., and supported, in part, with funds provided by the National Center for Research Resources, 5 M01 RR-01271 (Dr. Harmatz), 5 M01 RR-00400 (Dr. Whitley), M01 RR-00334 (Dr. Steiner), and UL1-RR-024134 (Dr. Kaplan). The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Center for Research Resources or the National Institutes of Health.
The MPS VI Study Group co-investigators are: John Waterson, MD, PhD and Elio Gizzi, MD, Children’s Hospital & Research Center Oakland, Oakland, California; Yasmina Amraoui, MD, Children’s Hosp, University of Mainz, Germany; Bonito Victor, MD, Unidade de Doenças Metabólicas, Departamento Pediatria, Hospital de Sao João, Porto, Portugal; Javier Arroyo, MD, Hospital San Pedro de Alcantara, Hospital de día de Pediatría, Caceres, Spain; D.N. Bennett-Jones, MD, Consultant General & Renal Physician, Whitehaven, UK; Philippe Bernard, MD, Centre Hospitalier d’Arras, Arras, France; Prof. Billette de Villemeur, Hôpital Trousseau, Paris, France; Raquel Boy, MD, Hospital Universitário Pedro Ernesto, Rio de Janeiro, Brazil; Eduardo Coopman, MD, Hospital del Cobre De. Salvador, Calama, Chile; Prof. Rudolf Korinthenberg, Universitätsklinikum Freiburg, Zentrum für Kinderheilkunde und Jugendmedizin, Klinik II Neuropädiatrie und Muskelerkrankungen, Freiburg, Germany; Michel Kretz, MD, Hôpital Civil de Colmar, Le Parc Centre de la Mère et de l’Enfant, Colmar, France; Shuan-Pei Lin, MD, MacKay Memorial Hospital, Department of Genetics, Taipei, Taiwan; Ana Maria Martins, MD, UNIFESP, Instituto de Oncologia Pediátrica, GRAACC/UNIFESP, Departamento de Pediatria, São Paulo, Brazil; Anne O’Meara, MD, Our Lady’s Hospital for Sick Children, Dublin, Ireland; Gregory Pastores, MD, PhD, NYU Medical Center, Rusk Institute, New York, New York; Lorenzo Pavone, MD, Rita Barone, MD, Agata Fiumara, MD, and Prof. Giovanni Sorge, Department of Pediatrics, University of Catania, Catania, Italy; Silvio Pozzi, MD, Ospedale Vito Fazzi, UO Pediatria, Lecce, Italy; Uwe Preiss, MD, Universitätsklinik und Poliklinik fűr Kinder, Halle, Germany; Emerson Santana Santos, MD, Fundação Universidade de Ciências da Saúde de Alagoas Governador, Departamento de Pediatria, Maceió, Brazil; Isabel Cristina Neves de Souza, MD and Luiz Carlos Santana da Silva, PhD, Universidade Federal do Pará, Centro de Ciências Biológicas, Hospital Universitário João de Barros Barreto, Belém, Brazil; Eugênia Ribeiro Valadares, MD, PhD, Hospital das Clínicas, Faculdade de Medicina da Universidade Federal de Minas Gerais-UFMG, Avenida Professor Alfredo Balena, Belo Horizonte-Minas Gerais, Brazil; Laura Keppen, MD, Department of Pediatrics, University of South Dakota School of Medicine, Sioux Falls, SD; David Sillence, MD, Children’s Hospital, Westmead, Australia; Lionel Lubitz, MD, Royal Children’s Hospital, Melbourne, Australia; William Frischman, MD, The Townsville Hospital, Townsville, Australia; Julie Simon, RN, Children’s Hospital & Research Center Oakland, Oakland, California; Claudia Lee, MPH, Children’s Hospital & Research Center Oakland, Oakland, California; Stephanie Oates, RN Department of Genetic Medicine, Women’s and Children’s Hospital Adelaide, North Adelaide, Australia; Lewis Waber, MD, PhD, Pediatric Genetics and Metabolism, University of Texas Southwest Medical Center, Dallas, TX; Ray Pais, MD, Pediatric Hematology/Oncology, East Tennessee Children’s Hospital, Knoxville, TN
Conflict of interest
Drs. Harmatz, Beck, Giugliani, Berger, Steiner, and Yu have provided consulting support to BioMarin Pharmaceutical Inc, Novato, CA, USA. Dr. Hopwood has received commercial research project funding to assist the development of enzyme replacement therapy for MPS VI patients. Drs. Harmatz and Arash each report receiving a speaker′s honorarium and travel support from BioMarin. BioMarin is a supporter of the Lysosomal Disease Network′s WORLD Symposium organized by Dr. Whitley. Drs. Swiedler and Decker are former and current employees of BioMarin Pharmaceutical, Inc., respectively; both are stockholders.
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