{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,4]],"date-time":"2026-05-04T01:13:58Z","timestamp":1777857238507,"version":"3.51.4"},"reference-count":41,"publisher":"SAGE Publications","issue":"4","license":[{"start":{"date-parts":[[2017,10,24]],"date-time":"2017-10-24T00:00:00Z","timestamp":1508803200000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Neuromuscular Diseases"],"published-print":{"date-parts":[[2017,10,24]]},"abstract":"<jats:sec>\n                    <jats:title>Background:<\/jats:title>\n                    <jats:p>Recent short-term clinical trials in patients with Duchenne Muscular Dystrophy (DMD) have indicated greater disease variability in terms of progression than expected. In addition, as average life-expectancy increases, reliable data is required on clinical progression in the older DMD population.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objective:<\/jats:title>\n                    <jats:p>To determine the effects of corticosteroids on major clinical outcomes of DMD in a large multinational cohort of genetically confirmed DMD patients.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>In this cross-sectional study we analysed clinical data from 5345 genetically confirmed DMD patients from 31 countries held within the TREAT-NMD global DMD database. For analysis patients were categorised by corticosteroid background and further stratified by age.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>Loss of ambulation in non-steroid treated patients was 10 years and in corticosteroid treated patients 13 years old ( p\u200a=\u200a0.0001). Corticosteroid treated patients were less likely to need scoliosis surgery ( p\u200a&lt;\u200a0.001) or ventilatory support ( p\u200a&lt;\u200a0.001) and there was a mild cardioprotective effect of corticosteroids in the patient population aged 20 years and older ( p\u200a=\u200a0.0035). Patients with a single deletion of exon 45 showed an increased survival in contrast to other single exon deletions.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions:<\/jats:title>\n                    <jats:p>This study provides data on clinical outcomes of DMD across many healthcare settings and including a sizeable cohort of older patients. Our data confirm the benefits of corticosteroid treatment on ambulation, need for scoliosis surgery, ventilation and, to a lesser extent, cardiomyopathy. This study underlines the importance of data collection via patient registries and the critical role of multi-centre collaboration in the rare disease field.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.3233\/jnd-170280","type":"journal-article","created":{"date-parts":[[2017,11,10]],"date-time":"2017-11-10T10:23:39Z","timestamp":1510309419000},"page":"293-306","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":147,"title":["Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database"],"prefix":"10.1177","volume":"4","author":[{"given":"Za\u00efda","family":"Koeks","sequence":"first","affiliation":[{"name":"Leiden University Medical Center, Department of Neurology, Leiden, The Netherlands"}]},{"given":"Catherine L.","family":"Bladen","sequence":"additional","affiliation":[{"name":"John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK"}]},{"given":"David","family":"Salgado","sequence":"additional","affiliation":[{"name":"AP-HM, H\u00f4pital d\u2019Enfants de la Timone, D\u00e9partement de G\u00e9n\u00e9tique M\u00e9dicale et de Biologie Cellulaire, Marseille, France"}]},{"given":"Erik","family":"van Zwet","sequence":"additional","affiliation":[{"name":"Leiden University Medical Center, Department of Medical Statistics, Leiden, The Netherlands"}]},{"given":"Oksana","family":"Pogoryelova","sequence":"additional","affiliation":[{"name":"John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK"}]},{"given":"Grace","family":"McMacken","sequence":"additional","affiliation":[{"name":"John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK"}]},{"given":"Soledad","family":"Monges","sequence":"additional","affiliation":[{"name":"Hospital de Pediatr\u00eda J. P. Garrahan, Pichincha, Argentina"}]},{"given":"Maria E.","family":"Foncuberta","sequence":"additional","affiliation":[{"name":"Hospital de Pediatr\u00eda J. P. Garrahan, Pichincha, Argentina"}]},{"given":"Kyriaki","family":"Kekou","sequence":"additional","affiliation":[{"name":"Department of Medical Genetics, Medical School, University of Athens, Choremio Research Laboratory, St. Sophia\u2019s Children\u2019s Hospital Thinon and Levadia Goudi, Athens, Greece"}]},{"given":"Konstantina","family":"Kosma","sequence":"additional","affiliation":[{"name":"Department of Medical Genetics, Medical School, University of Athens, Choremio Research Laboratory, St. Sophia\u2019s Children\u2019s Hospital Thinon and Levadia Goudi, Athens, Greece"}]},{"given":"Hugh","family":"Dawkins","sequence":"additional","affiliation":[{"name":"Office of Population Health Genomics, Department of Health, Perth, WA, Australia"}]},{"given":"Leanne","family":"Lamont","sequence":"additional","affiliation":[{"name":"Office of Population Health Genomics, Department of Health, Perth, WA, Australia"}]},{"given":"Matthew I.","family":"Bellgard","sequence":"additional","affiliation":[{"name":"Centre for Comparative Genomics, Murdoch University, Murdoch, WA, Australia"}]},{"given":"Anna J.","family":"Roy","sequence":"additional","affiliation":[{"name":"WIV-ISP, Brussels, Belgium"}]},{"given":"Teodora","family":"Chamova","sequence":"additional","affiliation":[{"name":"Department of Neurology, Medical University-Sofia, Sofia, Bulgaria"}]},{"given":"Velina","family":"Guergueltcheva","sequence":"additional","affiliation":[{"name":"Clinic of Neurology, University Hospital Sofiamed, Sofia, Bulgaria"}]},{"given":"Sophelia","family":"Chan","sequence":"additional","affiliation":[{"name":"Department of Paediatrics and Adolescent Medicine, Queen Mary Hospital, University of Hong Kong, Hong Kong, China"}]},{"given":"Lawrence","family":"Korngut","sequence":"additional","affiliation":[{"name":"Department of Clinical Neurosciences and Hotchkiss Brain Institute, University of Calgary, South Health Campus, Calgary, AB, Canada"}]},{"given":"Craig","family":"Campbell","sequence":"additional","affiliation":[{"name":"Department of Paediatrics, Clinical Neurological Sciences & Epidemiology, Western University, London, ON, Canada"}]},{"given":"Yi","family":"Dai","sequence":"additional","affiliation":[{"name":"Department of Neurology, Peking Union Medical College Hospital, Peking Union Medical College and Chinese Academy of Medical Sciences, Beijing, China"}]},{"given":"Jen","family":"Wang","sequence":"additional","affiliation":[{"name":"China DMD Care and Support Association c\/o China Dolls, Xicheng district, China"}]},{"given":"Nina","family":"Bari\u0161i\u0107","sequence":"additional","affiliation":[{"name":"Division of Paediatric Neurology, University Hospital Centre Zagreb (KBC Zagreb) University of Zagreb Medical School, Zagreb, Croatia"}]},{"given":"Petr","family":"Brabec","sequence":"additional","affiliation":[{"name":"Institute for Biostatistic and Analyses, Masaryk University, Brno, Czech Republic"}]},{"given":"Jaana","family":"L\u00e4hdetie","sequence":"additional","affiliation":[{"name":"Department of Child Neurology, Turku University Central Hospital, Turku, Finland"}]},{"given":"Maggie C.","family":"Walter","sequence":"additional","affiliation":[{"name":"Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Munich, Germany"}]},{"given":"Olivia","family":"Schreiber-Katz","sequence":"additional","affiliation":[{"name":"Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Munich, Germany"}]},{"given":"Veronika","family":"Karcagi","sequence":"additional","affiliation":[{"name":"NIEH, Department of Molecular Genetics and Diagnostics, Budapest, Hungary"}]},{"given":"Marta","family":"Garami","sequence":"additional","affiliation":[{"name":"NIEH, Department of Molecular Genetics and Diagnostics, Budapest, Hungary"}]},{"given":"Agnes","family":"Herczegfalvi","sequence":"additional","affiliation":[{"name":"Semmelweis Medical University, II. Department of Paediatric Neurology, Budapest, Hungary"}]},{"given":"Venkatarman","family":"Viswanathan","sequence":"additional","affiliation":[{"name":"Kanchi Kamakoti CHILDS Trust and Apollo Children\u2019s Hospitals, Chennai, India"}]},{"given":"Farhad","family":"Bayat","sequence":"additional","affiliation":[{"name":"Pasteur Institute of Iran, Karaj complex, Tehran, Iran"}]},{"given":"Filippo","family":"Buccella","sequence":"additional","affiliation":[{"name":"Parent Project Onlus, Rome, Italy"}]},{"given":"Alessandra","family":"Ferlini","sequence":"additional","affiliation":[{"name":"Department of Reproduction and Growth, Department of Medical Sciences, OSPFE, University of Ferrara, Ferrara, Italy"}]},{"given":"En","family":"Kimura","sequence":"additional","affiliation":[{"name":"214-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan"}]},{"given":"Janneke C.","family":"van den Bergen","sequence":"additional","affiliation":[{"name":"Leiden University Medical Center, Department of Neurology, Leiden, The Netherlands"}]},{"given":"Miriam","family":"Rodrigues","sequence":"additional","affiliation":[{"name":"Department of Neurology, Auckland DHB, Auckland, New Zealand"}]},{"given":"Richard","family":"Roxburgh","sequence":"additional","affiliation":[{"name":"Department of Neurology, Auckland DHB, Auckland, New Zealand"}]},{"given":"Anna","family":"Lusakowska","sequence":"additional","affiliation":[{"name":"Department of Neurology, Medical University of Warsaw, Warsaw, Poland"}]},{"given":"Anna","family":"Kostera-Pruszczyk","sequence":"additional","affiliation":[{"name":"Department of Neurology, Medical University of Warsaw, Warsaw, Poland"}]},{"given":"Ros\u00e1rio","family":"Santos","sequence":"additional","affiliation":[{"name":"Centro de Gen\u00e9tica M\u00e9dica Jacinto Magalh\u00e3es, Porto, Portugal"}]},{"given":"Elena","family":"Neagu","sequence":"additional","affiliation":[{"name":"National Institute of Legal Medicine \u201cMina Minovici\u201d \u2013 Genetics Laboratory, Bucharest, Romania"}]},{"given":"Svetlana","family":"Artemieva","sequence":"additional","affiliation":[{"name":"Moscow Institute of Pediatrics, Moscow, Russia"}]},{"given":"Vedrana Milic","family":"Rasic","sequence":"additional","affiliation":[{"name":"Clinic for Neurology and Psychiatry for Children and Youth, Faculty of Medicine, University of Belgrade, Belgrade, Serbia"}]},{"given":"Dina","family":"Vojinovic","sequence":"additional","affiliation":[{"name":"Clinic for Neurology and Psychiatry for Children and Youth, Faculty of Medicine, University of Belgrade, Belgrade, Serbia"},{"name":"Department of Epidemiology, Erasmus University, Medical Centre, Rotterdam, The Netherlands"}]},{"given":"Manuel","family":"Posada","sequence":"additional","affiliation":[{"name":"Institute of Rare Diseases Research, SpainRDR and CIBERER, Institute of Health Carlos III, Madrid, Spain"}]},{"given":"Clemens","family":"Bloetzer","sequence":"additional","affiliation":[{"name":"Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland"}]},{"given":"Andrea","family":"Klein","sequence":"additional","affiliation":[{"name":"Paediatric Neurology and Neurorehabilitation Unit, Lausanne University Hospital, Lausanne, Switzerland"}]},{"given":"Jordi","family":"D\u00edaz-Manera","sequence":"additional","affiliation":[{"name":"Unitat de Malalties Neuromusculars, Servei de Neurologia, Hospital de la Santa Creu i Sant Pau de Barcelona, Barcelona, Spain"}]},{"given":"Eduard","family":"Gallardo","sequence":"additional","affiliation":[{"name":"Paediatric Neurology and Neurorehabilitation Unit, Lausanne University Hospital, Lausanne, Switzerland"}]},{"given":"A. Ay\u015fe","family":"Karaduman","sequence":"additional","affiliation":[{"name":"Hacettepe University Faculty of Health Sciences Department of Physiotherapy and Rehabilitation, Altinda\u011f, Ankara, Turkey"}]},{"given":"Tunca","family":"Oznur","sequence":"additional","affiliation":[{"name":"Hacettepe University Faculty of Health Sciences Department of Physiotherapy and Rehabilitation, Altinda\u011f, Ankara, Turkey"}]},{"given":"Haluk","family":"Topalo\u011flu","sequence":"additional","affiliation":[{"name":"Hacettepe University Faculty of Health Sciences Department of Physiotherapy and Rehabilitation, Altinda\u011f, Ankara, Turkey"}]},{"given":"Rasha","family":"El Sherif","sequence":"additional","affiliation":[{"name":"Neurology & Neurogenic Unit, Egypt Air Hospital, Ain Shams University, Egypt"}]},{"given":"Angela","family":"Stringer","sequence":"additional","affiliation":[{"name":"Action Duchenne, Epicentre, London, UK"}]},{"given":"Andriy V.","family":"Shatillo","sequence":"additional","affiliation":[{"name":"Institute of Neurology, Psychiatry and Narcology of NAMS, Kharkiv, Ukraine"}]},{"given":"Ann S.","family":"Martin","sequence":"additional","affiliation":[{"name":"DuchenneConnect, Hackensack, NJ, USA"}]},{"given":"Holly L.","family":"Peay","sequence":"additional","affiliation":[{"name":"DuchenneConnect, Hackensack, NJ, USA"}]},{"given":"Jan","family":"Kirschner","sequence":"additional","affiliation":[{"name":"University Medical Center Freiburg, Freiburg, Germany"}]},{"given":"Kevin M.","family":"Flanigan","sequence":"additional","affiliation":[{"name":"Center for Gene Therapy, The Research Institute, Nationwide Children\u2019s Hospital, Columbus, OH, USA"}]},{"given":"Volker","family":"Straub","sequence":"additional","affiliation":[{"name":"John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK"}]},{"given":"Kate","family":"Bushby","sequence":"additional","affiliation":[{"name":"John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK"}]},{"given":"Christophe","family":"B\u00e9roud","sequence":"additional","affiliation":[{"name":"AP-HM, H\u00f4pital d\u2019Enfants de la Timone, D\u00e9partement de G\u00e9n\u00e9tique M\u00e9dicale et de Biologie Cellulaire, Marseille, France"}]},{"given":"Jan J.","family":"Verschuuren","sequence":"additional","affiliation":[{"name":"Leiden University Medical Center, Department of Neurology, Leiden, The Netherlands"}]},{"given":"Hanns","family":"Lochm\u00fcller","sequence":"additional","affiliation":[{"name":"John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK"}]}],"member":"179","published-online":{"date-parts":[[2017,11,10]]},"reference":[{"key":"ref001","doi-asserted-by":"publisher","DOI":"10.1002\/humu.22390"},{"key":"ref002","doi-asserted-by":"publisher","DOI":"10.1002\/humu.22758"},{"key":"ref003","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0108205"},{"key":"ref004","doi-asserted-by":"publisher","DOI":"10.1002\/mus.23808"},{"key":"ref005","doi-asserted-by":"publisher","DOI":"10.1002\/ana.23528"},{"key":"ref006","unstructured":"GowersWR, TaylorJ. 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