Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database

Author:

Koeks Zaïda1,Bladen Catherine L.2,Salgado David3,van Zwet Erik4,Pogoryelova Oksana2,McMacken Grace2,Monges Soledad5,Foncuberta Maria E.5,Kekou Kyriaki6,Kosma Konstantina6,Dawkins Hugh7,Lamont Leanne7,Bellgard Matthew I.8,Roy Anna J.9,Chamova Teodora10,Guergueltcheva Velina11,Chan Sophelia12,Korngut Lawrence13,Campbell Craig14,Dai Yi15,Wang Jen16,Barišić Nina17,Brabec Petr18,Lähdetie Jaana19,Walter Maggie C.20,Schreiber-Katz Olivia20,Karcagi Veronika21,Garami Marta21,Herczegfalvi Agnes22,Viswanathan Venkatarman23,Bayat Farhad24,Buccella Filippo25,Ferlini Alessandra26,Kimura En27,van den Bergen Janneke C.1,Rodrigues Miriam28,Roxburgh Richard28,Lusakowska Anna29,Kostera-Pruszczyk Anna29,Santos Rosário30,Neagu Elena31,Artemieva Svetlana32,Rasic Vedrana Milic33,Vojinovic Dina3334,Posada Manuel35,Bloetzer Clemens36,Klein Andrea37,Díaz-Manera Jordi38,Gallardo Eduard37,Karaduman A. Ayşe39,Oznur Tunca39,Topaloğlu Haluk39,El Sherif Rasha40,Stringer Angela41,Shatillo Andriy V.42,Martin Ann S.43,Peay Holly L.43,Kirschner Jan44,Flanigan Kevin M.45,Straub Volker2,Bushby Kate2,Béroud Christophe3,Verschuuren Jan J.1,Lochmüller Hanns2

Affiliation:

1. Leiden University Medical Center, Department of Neurology, Leiden, The Netherlands

2. John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Central Parkway, Newcastle upon Tyne, UK

3. AP-HM, Hôpital d’Enfants de la Timone, Département de Génétique Médicale et de Biologie Cellulaire, Marseille, France

4. Leiden University Medical Center, Department of Medical Statistics, Leiden, The Netherlands

5. Hospital de Pediatría J. P. Garrahan, Pichincha, Argentina

6. Department of Medical Genetics, Medical School, University of Athens, Choremio Research Laboratory, St. Sophia’s Children’s Hospital Thinon and Levadia Goudi, Athens, Greece

7. Office of Population Health Genomics, Department of Health, Perth, WA, Australia

8. Centre for Comparative Genomics, Murdoch University, Murdoch, WA, Australia

9. WIV-ISP, Brussels, Belgium

10. Department of Neurology, Medical University-Sofia, Sofia, Bulgaria

11. Clinic of Neurology, University Hospital Sofiamed, Sofia, Bulgaria

12. Department of Paediatrics and Adolescent Medicine, Queen Mary Hospital, University of Hong Kong, Hong Kong, China

13. Department of Clinical Neurosciences and Hotchkiss Brain Institute, University of Calgary, South Health Campus, Calgary, AB, Canada

14. Department of Paediatrics, Clinical Neurological Sciences & Epidemiology, Western University, London, ON, Canada

15. Department of Neurology, Peking Union Medical College Hospital, Peking Union Medical College and Chinese Academy of Medical Sciences, Beijing, China

16. China DMD Care and Support Association c/o China Dolls, Xicheng district, China

17. Division of Paediatric Neurology, University Hospital Centre Zagreb (KBC Zagreb) University of Zagreb Medical School, Zagreb, Croatia

18. Institute for Biostatistic and Analyses, Masaryk University, Brno, Czech Republic

19. Department of Child Neurology, Turku University Central Hospital, Turku, Finland

20. Friedrich-Baur-Institute, Department of Neurology, Ludwig-Maximilians-University of Munich, Munich, Germany

21. NIEH, Department of Molecular Genetics and Diagnostics, Budapest, Hungary

22. Semmelweis Medical University, II. Department of Paediatric Neurology, Budapest, Hungary

23. Kanchi Kamakoti CHILDS Trust and Apollo Children’s Hospitals, Chennai, India

24. Pasteur Institute of Iran, Karaj complex, Tehran, Iran

25. Parent Project Onlus, Rome, Italy

26. Department of Reproduction and Growth, Department of Medical Sciences, OSPFE, University of Ferrara, Ferrara, Italy

27. 214-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan

28. Department of Neurology, Auckland DHB, Auckland, New Zealand

29. Department of Neurology, Medical University of Warsaw, Warsaw, Poland

30. Centro de Genética Médica Jacinto Magalhães, Porto, Portugal

31. National Institute of Legal Medicine “Mina Minovici” – Genetics Laboratory, Bucharest, Romania

32. Moscow Institute of Pediatrics, Moscow, Russia

33. Clinic for Neurology and Psychiatry for Children and Youth, Faculty of Medicine, University of Belgrade, Belgrade, Serbia

34. Department of Epidemiology, Erasmus University, Medical Centre, Rotterdam, The Netherlands

35. Institute of Rare Diseases Research, SpainRDR and CIBERER, Institute of Health Carlos III, Madrid, Spain

36. Institute of Social and Preventive Medicine, University of Bern, Bern, Switzerland

37. Paediatric Neurology and Neurorehabilitation Unit, Lausanne University Hospital, Lausanne, Switzerland

38. Unitat de Malalties Neuromusculars, Servei de Neurologia, Hospital de la Santa Creu i Sant Pau de Barcelona, Barcelona, Spain

39. Hacettepe University Faculty of Health Sciences Department of Physiotherapy and Rehabilitation, Altindağ, Ankara, Turkey

40. Neurology & Neurogenic Unit, Egypt Air Hospital, Ain Shams University, Egypt

41. Action Duchenne, Epicentre, London, UK

42. Institute of Neurology, Psychiatry and Narcology of NAMS, Kharkiv, Ukraine

43. DuchenneConnect, Hackensack, NJ, USA

44. University Medical Center Freiburg, Freiburg, Germany

45. Center for Gene Therapy, The Research Institute, Nationwide Children’s Hospital, Columbus, OH, USA

Publisher

IOS Press

Subject

Neurology (clinical),Neurology

Cited by 114 articles. 订阅此论文施引文献 订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献

同舟云学术

1.学者识别学者识别

2.学术分析学术分析

3.人才评估人才评估

"同舟云学术"是以全球学者为主线,采集、加工和组织学术论文而形成的新型学术文献查询和分析系统,可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容,当前同舟云学术共收录了国内外主流学术期刊6万余种,收集的期刊论文及会议论文总量共计约1.5亿篇,并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询!咨询电话:010-8811{复制后删除}0370

www.globalauthorid.com

TOP

Copyright © 2019-2024 北京同舟云网络信息技术有限公司
京公网安备11010802033243号  京ICP备18003416号-3