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Safety and efficacy of tamoxifen in boys with Duchenne muscular dystrophy (TAMDMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
Henzi, Bettina C; Schmidt, Simone; Nagy, Sara; Rubino-Nacht, Daniela; Schaedelin, Sabine; Putananickal, Niveditha; Stimpson, Georgia; Amthor, Helge; Childs, Anne-Marie; Deconinck, Nicolas; de Groot, Imelda; Horrocks, Iain; Houwen-van Opstal, Saskia; Laugel, Vincent; Lopez Lobato, Mercedes; Madruga Garrido, Marcos; Nascimento Osorio, Andrés; Schara-Schmidt, Ulrike; Spinty, Stefan; von Moers, Arpad; Lawrence, Fiona; Hafner, Patricia; Dorchies, Olivier M; Fischer, Dirk.
Afiliação
  • Henzi BC; Division of Neuropediatrics and Developmental Medicine, University Children's Hospital Basel, University of Basel, Basel, Switzerland; Department of Neuropediatrics and Muscle Disorders, Medical Center-University of Freiburg, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
  • Schmidt S; Division of Neuropediatrics and Developmental Medicine, University Children's Hospital Basel, University of Basel, Basel, Switzerland.
  • Nagy S; Department of Neurology, University Hospital Basel, University of Basel, Basel, Switzerland.
  • Rubino-Nacht D; Division of Neuropediatrics and Developmental Medicine, University Children's Hospital Basel, University of Basel, Basel, Switzerland.
  • Schaedelin S; Department of Clinical Research, University of Basel and University Hospital Basel, Basel, Switzerland.
  • Putananickal N; Division of Neuropediatrics and Developmental Medicine, University Children's Hospital Basel, University of Basel, Basel, Switzerland.
  • Stimpson G; Developmental Neuroscience Research and Teaching Department, Faculty of Population Health Sciences, Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK.
  • Amthor H; Service de Neurologie et Réanimation Pédiatriques, APHP Paris Saclay, Hôpital Raymond Poincaré, Garches, France.
  • Childs AM; The Leeds Teaching Hospitals NHS Trust, Leeds, UK.
  • Deconinck N; Department of Paediatric Neurology and Neuromuscular Reference Center, Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels, Belgium.
  • de Groot I; Department of Rehabilitation, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, Nijmegen, Netherlands.
  • Horrocks I; Royal Hospital for Children, Glasgow, UK.
  • Houwen-van Opstal S; Department of Rehabilitation, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, Nijmegen, Netherlands.
  • Laugel V; Department of Pediatric Neurology, Strasbourg University Hospital, Strasbourg, France.
  • Lopez Lobato M; Sección de Neurología Pediátrica, Hospital Universitario Virgen del Rocío, Seville, Spain.
  • Madruga Garrido M; Sección de Neurología Pediátrica, Hospital Universitario Virgen del Rocío, Seville, Spain.
  • Nascimento Osorio A; Neuromuscular Unit, Department of Neurology, Hospital Sant Joan de Déu and Center for Biomedical Research Network on Rare Diseases, ISCIII, Barcelona, Spain.
  • Schara-Schmidt U; Department of Pediatric Neurology, University Hospital Essen, University of Duisburg-Essen, Essen, Germany.
  • Spinty S; Alder Hey Children's Hospital, Liverpool, UK.
  • von Moers A; Department of Pediatrics, DRK Kliniken Berlin Westend, Berlin, Germany.
  • Lawrence F; Duchenne UK, London, UK.
  • Hafner P; Division of Neuropediatrics and Developmental Medicine, University Children's Hospital Basel, University of Basel, Basel, Switzerland.
  • Dorchies OM; School of Pharmaceutical Sciences and Institute of Pharmaceutical Sciences of Western Switzerland, University of Geneva, Geneva, Switzerland.
  • Fischer D; Division of Neuropediatrics and Developmental Medicine, University Children's Hospital Basel, University of Basel, Basel, Switzerland. Electronic address: dirk.fischer@ukbb.ch.
Lancet Neurol ; 22(10): 890-899, 2023 10.
Article em En | MEDLINE | ID: mdl-37739572

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Distrofia Muscular de Duchenne Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Animals / Humans / Male Idioma: En Revista: Lancet Neurol Assunto da revista: NEUROLOGIA Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Alemanha

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Distrofia Muscular de Duchenne Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Animals / Humans / Male Idioma: En Revista: Lancet Neurol Assunto da revista: NEUROLOGIA Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Alemanha