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Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
Guglieri, Michela; Clemens, Paula R; Perlman, Seth J; Smith, Edward C; Horrocks, Iain; Finkel, Richard S; Mah, Jean K; Deconinck, Nicolas; Goemans, Nathalie; Haberlova, Jana; Straub, Volker; Mengle-Gaw, Laurel J; Schwartz, Benjamin D; Harper, Amy D; Shieh, Perry B; De Waele, Liesbeth; Castro, Diana; Yang, Michelle L; Ryan, Monique M; McDonald, Craig M; Tulinius, Mar; Webster, Richard; McMillan, Hugh J; Kuntz, Nancy L; Rao, Vashmi K; Baranello, Giovanni; Spinty, Stefan; Childs, Anne-Marie; Sbrocchi, Annie M; Selby, Kathryn A; Monduy, Migvis; Nevo, Yoram; Vilchez-Padilla, Juan J; Nascimento-Osorio, Andres; Niks, Erik H; de Groot, Imelda J M; Katsalouli, Marina; James, Meredith K; van den Anker, Johannes; Damsker, Jesse M; Ahmet, Alexandra; Ward, Leanne M; Jaros, Mark; Shale, Phil; Dang, Utkarsh J; Hoffman, Eric P.
Afiliação
  • Guglieri M; John Walton Muscular Dystrophy Research Centre, Newcastle Hospitals NHS Foundation Trust and Newcastle University, Newcastle, United Kingdom.
  • Clemens PR; University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania.
  • Perlman SJ; University of Washington School of Medicine, Seattle.
  • Smith EC; Duke University School of Medicine, Durham, North Carolina.
  • Horrocks I; Royal Hospital for Children, Glasgow, United Kingdom.
  • Finkel RS; Nemours Children's Hospital, Orlando, Florida.
  • Mah JK; St Jude Children's Research Hospital, Memphis, Tennessee.
  • Deconinck N; Alberta Children's Hospital Research Institute, University of Calgary, Calgary, Alberta, Canada.
  • Goemans N; Neuromuscular Reference Center, UZ Ghent, Ghent, Belgium.
  • Haberlova J; Department of Development and Regeneration, KU Leuven, Leuven, Belgium.
  • Straub V; Department of Paediatric Neurology, University Hospitals Leuven, Leuven, Belgium.
  • Mengle-Gaw LJ; Neuromuscular Centre, Department of Pediatric Neurology, Motol University Hospital, 2nd Medical School, Charles University, Prague, Czech Republic.
  • Schwartz BD; John Walton Muscular Dystrophy Research Centre, Newcastle Hospitals NHS Foundation Trust and Newcastle University, Newcastle, United Kingdom.
  • Harper AD; The Camden Group, St Louis, Missouri.
  • Shieh PB; The Camden Group, St Louis, Missouri.
  • De Waele L; Richmond Children's Hospital, Richmond, Virginia.
  • Castro D; UCLA Medical School, Los Angeles, California.
  • Yang ML; Department of Development and Regeneration, KU Leuven, Leuven, Belgium.
  • Ryan MM; Department of Paediatric Neurology, University Hospitals Leuven, Leuven, Belgium.
  • McDonald CM; UT Southwestern Medical Center, Dallas, Texas.
  • Tulinius M; University of Colorado School of Medicine, Children's Hospital Colorado, Aurora.
  • Webster R; The Royal Children's Hospital, Melbourne, Australia.
  • McMillan HJ; Murdoch Children's Research Institute, Melbourne, Australia.
  • Kuntz NL; University of California, Davis, Sacramento.
  • Rao VK; Queen Silvia Children's Hospital, Gothenburg, Sweden.
  • Baranello G; Kids Neuroscience Centre, The Children's Hospital at Westmead, Westmead, Australia.
  • Spinty S; University of Ottawa, Ottawa, Ontario, Canada.
  • Childs AM; Ann & Robert H. Lurie Children's Hospital, Chicago, Illinois.
  • Sbrocchi AM; Ann & Robert H. Lurie Children's Hospital, Chicago, Illinois.
  • Selby KA; The Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, London, United Kingdom.
  • Monduy M; Alder Hey Children's NHS Foundation Trust, Liverpool, United Kingdom.
  • Nevo Y; Leeds Teaching Hospitals Trust, Leeds, United Kingdom.
  • Vilchez-Padilla JJ; Montreal Children's Hospital, Montreal, Quebec, Canada.
  • Nascimento-Osorio A; BC Children's Hospital Research Institute, Vancouver, British Columbia, Canada.
  • Niks EH; Nemours Children's Hospital, Orlando, Florida.
  • de Groot IJM; Schneider Children's Medical Center, Tel Aviv University, Tel Aviv, Israel.
  • Katsalouli M; Hospital Quirónsalud Valencia, Valencia, Spain.
  • James MK; Neuropaediatrics Department, Institut de Recerca Pediàtrica Hospital Sant Joan de Déu, Barcelona, Spain.
  • van den Anker J; Leiden University Medical Center, Leiden, the Netherlands.
  • Damsker JM; UMC St Radboud, Nijmegen, the Netherlands.
  • Ahmet A; P&A Kyriakou Children's Hospital, Athens, Greece.
  • Ward LM; John Walton Muscular Dystrophy Research Centre, Newcastle Hospitals NHS Foundation Trust and Newcastle University, Newcastle, United Kingdom.
  • Jaros M; ReveraGen BioPharma, Rockville, Maryland.
  • Shale P; Children's National Medical Center, Washington, DC.
  • Dang UJ; ReveraGen BioPharma, Rockville, Maryland.
  • Hoffman EP; Children's Hospital of Eastern Ontario Research Institute, Ottawa, Ontario, Canada.
JAMA Neurol ; 79(10): 1005-1014, 2022 10 01.
Article em En | MEDLINE | ID: mdl-36036925
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Insuficiência Adrenal / Distrofia Muscular de Duchenne Tipo de estudo: Clinical_trials Limite: Child, preschool / Humans / Male Idioma: En Revista: JAMA Neurol Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Reino Unido
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Insuficiência Adrenal / Distrofia Muscular de Duchenne Tipo de estudo: Clinical_trials Limite: Child, preschool / Humans / Male Idioma: En Revista: JAMA Neurol Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Reino Unido