المؤلفون: Guglieri, M., Bushby, K., Mcdermott, M. P., Hart, K. A., Tawil, R., Martens, W. B., Herr, B. E., Mccoll, E., Speed, C., Wilkinson, J., Kirschner, J., King, W. M., Eagle, M., Brown, M. W., Willis, T., Griggs, R. C., Straub, V., van Ruiten, H., Childs, A. -M., Ciafaloni, E., Shieh, P. B., Spinty, S., Maggi, L., Baranello, G., Butterfield, R. J., Horrocks, I. A., Roper, H., Alhaswani, Z., Flanigan, K. M., Kuntz, N. L., Manzur, A., Darras, B. T., Kang, P. B., Morrison, L., Krzesniak-Swinarska, M., Mah, J. K., Mongini, T. E., Ricci, F., von der Hagen, M., Finkel, R. S., O'Reardon, K., Wicklund, M., Kumar, A., Mcdonald, C. M., Han, J. J., Joyce, N., Henricson, E. K., Schara-Schmidt, U., Gangfuss, A., Wilichowski, E., Barohn, R. J., Statland, J. M., Campbell, C., Vita, G., Vita, G. L., Howard, J. F., Hughes, I., Mcmillan, H. J., Pegoraro, E., Bello, L., Burnette, W. B., Thangarajh, M., Chang, T.

المساهمون: Schara-Schmidt, Ulrike (Beitragende*r), Gangfuss, Andrea (Beitragende*r)

المصدر: JAMA

مصطلحات موضوعية: Male, Medizin, General Medicine, Duchenne, Muscular Dystrophy, Duchenne, Child, Child, Preschool, Female, Humans, Pregnenediones, Glucocorticoids, Prednisone, Muscular Dystrophy, Preschool, Original Investigation

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0b1feacdba62af12b8e4e14d2e6f1136
https://www.ncbi.nlm.nih.gov/pubmed/35381069

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المؤلفون: Guglieri M; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, England., Bushby K; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, England., McDermott MP; Department of Neurology, University of Rochester Medical Center, Rochester, New York.; Department of Biostatistics and Computational Biology, University of Rochester Medical Center, Rochester, New York., Hart KA; Department of Neurology, University of Rochester Medical Center, Rochester, New York., Tawil R; Department of Neurology, University of Rochester Medical Center, Rochester, New York., Martens WB; Department of Neurology, University of Rochester Medical Center, Rochester, New York., Herr BE; Department of Neurology, University of Rochester Medical Center, Rochester, New York., McColl E; Newcastle University, Newcastle upon Tyne, England., Speed C; Newcastle University, Newcastle upon Tyne, England.; NIHR Clinical Research Network North East and North Cumbria, Newcastle upon Tyne, England., Wilkinson J; Newcastle University, Newcastle upon Tyne, England., Kirschner J; Department of Neuropediatrics and Muscle Disorders, Faculty of Medicine, University Hospital Freiburg, Freiburg, Germany.; Department of Neuropediatrics, University Hospital Bonn, Bonn, Germany., King WM; Ohio State University, Columbus., Eagle M; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, England., Brown MW; Department of Neurology, University of Rochester Medical Center, Rochester, New York., Willis T; Robert Jones and Agnes Hunt Orthopaedic Hospital, NHS Foundation Trust, Oswestry, England., Griggs RC; Department of Neurology, University of Rochester Medical Center, Rochester, New York., Straub V; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, England., van Ruiten H; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, England., Childs AM; Leeds General Infirmary, Leeds, England., Ciafaloni E; Department of Neurology, University of Rochester Medical Center, Rochester, New York., Shieh PB; David Geffen School of Medicine, University of California, Los Angeles., Spinty S; Alderhey Children's Hospital NHS Foundation Trust, Liverpool, England., Maggi L; Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy., Baranello G; Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.; Dubowitz Neuromuscular Centre, UCL NIHR GOSH Biomedical Research Centre, UCL Great Ormond Street Institute of Child Health, London, England., Butterfield RJ; Department of Pediatric Neurology, University of Utah, Salt Lake City., Horrocks IA; Glasgow Paediatric Neuromuscular Research Centre, Greater Glasgow and Clyde NHS Yorkhill Hospital, Glasgow, Scotland., Roper H; University Hospitals Birmingham NHS Foundation Trust, Birmingham, England., Alhaswani Z; University Hospitals Birmingham NHS Foundation Trust, Birmingham, England., Flanigan KM; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, Ohio., Kuntz NL; Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois., Manzur A; Great Ormond Street Hospital, London, England., Darras BT; Department of Neurology, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts., Kang PB; Department of Neurology, Boston Children's Hospital and Harvard Medical School, Boston, Massachusetts.; Paul and Sheila Wellstone Muscular Dystrophy Center and Department of Neurology, University of Minnesota Medical School, Minneapolis., Morrison L; Health Sciences Center, University of New Mexico, Albuquerque., Krzesniak-Swinarska M; Health Sciences Center, University of New Mexico, Albuquerque., Mah JK; Cumming School of Medicine, University of Calgary and Alberta Children's Hospital Research Institute, Calgary, Canada., Mongini TE; Neuromuscular Center, AOU Città della Salute e della Scienza, University of Turin, Turin, Italy., Ricci F; Neuromuscular Center, AOU Città della Salute e della Scienza, University of Turin, Turin, Italy., von der Hagen M; Abteilung Neuropädiatrie, Medizinische Fakultät Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany., Finkel RS; Nemours Children's Hospital, Orlando, Florida.; Center for Experimental Neurotherapeutics, St Jude Children's Research Hospital, Memphis, Tennessee., O'Reardon K; Nemours Children's Hospital, Orlando, Florida., Wicklund M; Penn State Health Milton S. Hershey Medical Center, Hershey, Pennsylvania.; Department of Neurology, School of Medicine, University of Colorado, Aurora., Kumar A; Penn State Health Milton S. Hershey Medical Center, Hershey, Pennsylvania., McDonald CM; Department of Physical Medicine and Rehabilitation and Department of Pediatrics, University of California-Davis, Sacramento., Han JJ; Department of Physical Medicine and Rehabilitation and Department of Pediatrics, University of California-Davis, Sacramento., Joyce N; Department of Physical Medicine and Rehabilitation and Department of Pediatrics, University of California-Davis, Sacramento., Henricson EK; Department of Physical Medicine and Rehabilitation and Department of Pediatrics, University of California-Davis, Sacramento., Schara-Schmidt U; Clinic for Pediatrics I, Pediatric Neurology, University Hospital Essen, Essen, Germany., Gangfuss A; Clinic for Pediatrics I, Pediatric Neurology, University Hospital Essen, Essen, Germany., Wilichowski E; Department of Pediatrics and Adolescent Medicine, Division of Pediatric Neurology, University Medical Center Göttingen, Göttingen, Germany., Barohn RJ; University of Missouri, Columbia., Statland JM; University of Kansas Medical Center, Kansas City., Campbell C; Departments of Pediatrics, Clinical Neurological Sciences, and Epidemiology, University of Western Ontario, London, Canada., Vita G; ERN Neuromuscular Unit, Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy., Vita GL; Unit of Neurology, IRCCS Centro Neurolesi Bonino-Pulejo, Messina, Italy., Howard JF Jr; School of Medicine, University of North Carolina, Chapel Hill., Hughes I; Royal Manchester Children's Hospital, Manchester, England., McMillan HJ; Children's Hospital of Eastern Ontario Research Institute, University of Ottawa, Ottawa, Canada., Pegoraro E; ERN Neuromuscular Unit, Department of Neuroscience, University of Padova, Padua, Italy., Bello L; ERN Neuromuscular Unit, Department of Neuroscience, University of Padova, Padua, Italy., Burnette WB; Vanderbilt University Medical Center, Nashville, Tennessee., Thangarajh M; Department of Neurology, Virginia Commonwealth University, Richmond., Chang T; Children's National Hospital, George Washington University, Washington, DC.

مؤلفون مشاركون: FOR-DMD Investigators of the Muscle Study Group

المصدر: JAMA [JAMA] 2022 Apr 19; Vol. 327 (15), pp. 1456-1468.

نوع المنشور: Comparative Study; Journal Article; Randomized Controlled Trial

بيانات الدورية: Publisher: American Medical Association Country of Publication: United States NLM ID: 7501160 Publication Model: Print Cited Medium: Internet ISSN: 1538-3598 (Electronic) Linking ISSN: 00987484 NLM ISO Abbreviation: JAMA Subsets: MEDLINE

مواضيع طبية MeSH: Glucocorticoids*/administration & dosage , Glucocorticoids*/adverse effects , Glucocorticoids*/therapeutic use , Muscular Dystrophy, Duchenne*/drug therapy , Prednisone*/administration & dosage , Prednisone*/adverse effects , Prednisone*/therapeutic use, Child ; Child, Preschool ; Female ; Humans ; Male ; Pregnenediones/adverse effects