Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database
- Koeks, Zaïda Leiden University Medical Center, Department of Neurology, Leiden, The Netherlands
- Bladen, Catherine L. John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK
- Salgado, David AP-HM, Hôpital d’Enfants de la Timone, Département de Génétique Médicale et de Biologie Cellulaire, Marseille, France
- van Zwet, Erik Leiden University Medical Center, Department of Medical Statistics, Leiden, The Netherlands
- Pogoryelova, Oksana John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK
- McMacken, Grace John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK
- Monges, Soledad Hospital de Pediatría J. P. Garrahan, Pichincha, Argentina
- Foncuberta, Maria E. Hospital de Pediatría J. P. Garrahan, Pichincha, Argentina
- Kekou, Kyriaki Department of Medical Genetics, Medical School, University of Athens, Choremio Research Laboratory, St. Sophia’s Children’s Hospital Thinon and Levadia Goudi, Athens, Greece
- Kosma, Konstantina Department of Medical Genetics, Medical School, University of Athens, Choremio Research Laboratory, St. Sophia’s Children’s Hospital Thinon and Levadia Goudi, Athens, Greece
- Dawkins, Hugh Office of Population Health Genomics, Department of Health, Perth, WA, Australia
- Lamont, Leanne Office of Population Health Genomics, Department of Health, Perth, WA, Australia
- Bellgard, Matthew I. Centre for Comparative Genomics, Murdoch University, Murdoch, WA, Australia
- Roy, Anna J. WIV-ISP, Brussels, Belgium
- Chamova, Teodora Department of Neurology, Medical University-Sofia, Sofia, Bulgaria
- Guergueltcheva, Velina Clinic of Neurology, University Hospital Sofiamed, Sofia, Bulgaria
- Chan, Sophelia Department of Paediatrics and Adolescent Medicine, Queen Mary Hospital, University of Hong Kong, Hong Kong, China
- Korngut, Lawrence Department of Clinical Neurosciences and Hotchkiss Brain Institute, University of Calgary, South Health Campus, Calgary, AB, Canada
- Campbell, Craig Department of Paediatrics, Clinical Neurological Sciences & Epidemiology, Western University, London, ON, Canada
- Dai, Yi Department of Neurology, Peking Union Medical College Hospital, Peking Union Medical College and Chinese Academy of Medical Sciences, Beijing, China
- Wang, Jen China DMD Care and Support Association c/o China Dolls, Xicheng district, China
- Barišić, Nina Division of Paediatric Neurology, University Hospital Centre Zagreb (KBC Zagreb) University of Zagreb Medical School, Zagreb, Croatia
- Brabec, Petr Institute for Biostatistic and Analyses, Masaryk University, Brno, Czech Republic
- Lähdetie, Jaana Department of Child Neurology, Turku University Central Hospital, Turku, Finland
- Walter, Maggie C. Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Munich, Germany
- Schreiber-Katz, Olivia Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Munich, Germany
- Karcagi, Veronika NIEH, Department of Molecular Genetics and Diagnostics, Budapest, Hungary
- Garami, Marta NIEH, Department of Molecular Genetics and Diagnostics, Budapest, Hungary
- Herczegfalvi, Agnes Semmelweis Medical University, II. Department of Paediatric Neurology, Budapest, Hungary
- Viswanathan, Venkatarman Kanchi Kamakoti CHILDS Trust and Apollo Children’s Hospitals, Chennai, India
- Bayat, Farhad Pasteur Institute of Iran, Karaj complex, Tehran, Iran
- Buccella, Filippo Parent Project Onlus, Rome, Italy
- Ferlini, Alessandra Department of Reproduction and Growth, Department of Medical Sciences, OSPFE, University of Ferrara, Ferrara, Italy
- Kimura, En 214-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan
- van den Bergen, Janneke C. Leiden University Medical Center, Department of Neurology, Leiden, The Netherlands
- Rodrigues, Miriam Department of Neurology, Auckland DHB, Auckland, New Zealand
- Roxburgh, Richard Department of Neurology, Auckland DHB, Auckland, New Zealand
- Lusakowska, Anna Department of Neurology, Medical University of Warsaw, Warsaw, Poland
- Kostera-Pruszczyk, Anna Department of Neurology, Medical University of Warsaw, Warsaw, Poland
- Santos, Rosário Centro de Genética Médica Jacinto Magalhães, Porto, Portugal
- Neagu, Elena National Institute of Legal Medicine “Mina Minovici” – Genetics Laboratory, Bucharest, Romania
- Artemieva, Svetlana Moscow Institute of Pediatrics, Moscow, Russia
- Rasic, Vedrana Milic Clinic for Neurology and Psychiatry for Children and Youth, Faculty of Medicine, University of Belgrade, Belgrade, Serbia
- Vojinovic, Dina Department of Epidemiology, Erasmus University, Medical Centre, Rotterdam, The Netherlands
- Posada, Manuel Institute of Rare Diseases Research, SpainRDR and CIBERER, Institute of Health Carlos III, Madrid, Spain
- Bloetzer, Clemens Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland
- Klein, Andrea Paediatric Neurology and Neurorehabilitation Unit, Lausanne University Hospital, Lausanne, Switzerland
- Díaz-Manera, Jordi Unitat de Malalties Neuromusculars, Servei de Neurologia, Hospital de la Santa Creu i Sant Pau de Barcelona, Barcelona, Spain
- Gallardo, Eduard Paediatric Neurology and Neurorehabilitation Unit, Lausanne University Hospital, Lausanne, Switzerland
- Karaduman, A. Ayşe Hacettepe University Faculty of Health Sciences Department of Physiotherapy and Rehabilitation, Altindağ, Ankara, Turkey
- Oznur, Tunca Hacettepe University Faculty of Health Sciences Department of Physiotherapy and Rehabilitation, Altindağ, Ankara, Turkey
- Topaloğlu, Haluk Hacettepe University Faculty of Health Sciences Department of Physiotherapy and Rehabilitation, Altindağ, Ankara, Turkey
- El Sherif, Rasha Neurology & Neurogenic Unit, Egypt Air Hospital, Ain Shams University, Egypt
- Stringer, Angela Action Duchenne, Epicentre, London, UK
- Shatillo, Andriy V. Institute of Neurology, Psychiatry and Narcology of NAMS, Kharkiv, Ukraine
- Martin, Ann S. DuchenneConnect, Hackensack, NJ, USA
- Peay, Holly L. DuchenneConnect, Hackensack, NJ, USA
- Kirschner, Jan University Medical Center Freiburg, Freiburg, Germany
- Flanigan, Kevin M. Center for Gene Therapy, The Research Institute, Nationwide Children’s Hospital, Columbus, OH, USA
- Straub, Volker John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK
- Bushby, Kate John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK
- Béroud, Christophe AP-HM, Hôpital d’Enfants de la Timone, Département de Génétique Médicale et de Biologie Cellulaire, Marseille, France
- Verschuuren, Jan J. Leiden University Medical Center, Department of Neurology, Leiden, The Netherlands
- Lochmüller, Hanns John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK
Published in:
- Journal of Neuromuscular Diseases. - IOS Press. - 2017, vol. 4, no. 4, p. 293-306
- Language
-
- English
- Open access status
- bronze
- Identifiers
-
- DOI 10.3233/jnd-170280
- ISSN 2214-3599
- Persistent URL
- https://sonar.ch/global/documents/239680
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