Safety and efficacy of tamoxifen in boys with Duchenne muscular dystrophy (TAMDMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.

Autor: 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.

Publikováno v: The Lancet. Neurology [Lancet Neurol] 2023 Oct; Vol. 22 (10), pp. 890-899.

Health-related quality of life, self-reported impairments and activities of daily living in relation to muscle function in post-polio syndrome.

Autor: Gocheva V; Division of Neuropediatrics and Developmental Medicine, University Children's Hospital of Basel (UKBB),University of Basel, Spitalstrasse 33, Postfach, 4056, Basel, Switzerland. vanya.gocheva@ukbb.ch., Hafner P; Division of Neuropediatrics and Developmental Medicine, University Children's Hospital of Basel (UKBB),University of Basel, Spitalstrasse 33, Postfach, 4056, Basel, Switzerland., Orsini AL; Division of Neuropediatrics and Developmental Medicine, University Children's Hospital of Basel (UKBB),University of Basel, Spitalstrasse 33, Postfach, 4056, Basel, Switzerland.; Division of Neurology, University Hospital Basel, Basel, Switzerland., Schmidt S; Division of Neuropediatrics and Developmental Medicine, University Children's Hospital of Basel (UKBB),University of Basel, Spitalstrasse 33, Postfach, 4056, Basel, Switzerland.; Department of Neurology, General Hospital Hietzing with Neurological Center Rosenhügel, Vienna, Austria., Schaedelin S; Department of Clinical Research, Clinical Trial Unit, University Hospital Basel, Basel, Switzerland., Rueedi N; Division of Neuropediatrics and Developmental Medicine, University Children's Hospital of Basel (UKBB),University of Basel, Spitalstrasse 33, Postfach, 4056, Basel, Switzerland., Rubino-Nacht D; Division of Neuropediatrics and Developmental Medicine, University Children's Hospital of Basel (UKBB),University of Basel, Spitalstrasse 33, Postfach, 4056, Basel, Switzerland., Weber P; Division of Neuropediatrics and Developmental Medicine, University Children's Hospital of Basel (UKBB),University of Basel, Spitalstrasse 33, Postfach, 4056, Basel, Switzerland., Fischer D; Division of Neuropediatrics and Developmental Medicine, University Children's Hospital of Basel (UKBB),University of Basel, Spitalstrasse 33, Postfach, 4056, Basel, Switzerland.

Publikováno v: Journal of patient-reported outcomes [J Patient Rep Outcomes] 2020 Jul 16; Vol. 4 (1), pp. 59. Date of Electronic Publication: 2020 Jul 16.

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Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy.

Autor: Nagy S; Division of Pediatric Neurology, University Children's Hospital Basel (UKBB), University of Basel; Department of Neurology, University Hospital Basel, University of Basel; sara.nagy@ukbb.ch., Schmidt S; Division of Pediatric Neurology, University Children's Hospital Basel (UKBB), University of Basel; Department of Neurology, University Hospital Basel, University of Basel., Hafner P; Division of Pediatric Neurology, University Children's Hospital Basel (UKBB), University of Basel; Division of Neurology, University Clinic of Medicine, Cantonal Hospital Baselland, Bruderholz., Klein A; Division of Pediatric Neurology, University Children's Hospital Basel (UKBB), University of Basel; Division of Pediatric Neurology, CHUV; Division of Neuropaediatrics, Inselspital, University Children's Hospital Bern., Rubino-Nacht D; Division of Pediatric Neurology, University Children's Hospital Basel (UKBB), University of Basel., Gocheva V; Division of Pediatric Neurology, University Children's Hospital Basel (UKBB), University of Basel., Bieri O; Department of Radiology, Division of Radiological Physics, University Hospital Basel, University of Basel., Vuillerot C; Hospices Civils de Lyon, Hôpital Femme-Mère-Enfant, L'Escale, Service de Médecine Physique et de Réadaptation Pédiatrique., Bonati U; Division of Pediatric Neurology, University Children's Hospital Basel (UKBB), University of Basel; Department of Neurology, University Hospital Basel, University of Basel., Fischer D; Division of Pediatric Neurology, University Children's Hospital Basel (UKBB), University of Basel; Department of Neurology, University Hospital Basel, University of Basel; Division of Neurology, University Clinic of Medicine, Cantonal Hospital Baselland, Bruderholz.

Publikováno v: Journal of visualized experiments : JoVE [J Vis Exp] 2019 Jan 12 (143). Date of Electronic Publication: 2019 Jan 12.

Timed function tests, motor function measure, and quantitative thigh muscle MRI in ambulant children with Duchenne muscular dystrophy: A cross-sectional analysis.

Autor: Schmidt S; Division of Pediatric Neurology, University of Basel Children's Hospital, Basel, Switzerland; Department of Neurology, University of Basel Hospital, Basel, Switzerland., Hafner P; Division of Pediatric Neurology, University of Basel Children's Hospital, Basel, Switzerland; Division of Neurology, Medical University Clinic, Kantonsspital Baselland, Bruderholz, Switzerland., Klein A; Division of Pediatric Neurology, University of Basel Children's Hospital, Basel, Switzerland; Division of Pediatric Neurology, Lausanne University Hospital, Lausanne, Switzerland; Division of Pediatric Neurology, University of Berne Hospital, Berne, Switzerland., Rubino-Nacht D; Division of Pediatric Neurology, University of Basel Children's Hospital, Basel, Switzerland., Gocheva V; Division of Pediatric Neurology, University of Basel Children's Hospital, Basel, Switzerland., Schroeder J; Division of Pediatric Neurology, University of Basel Children's Hospital, Basel, Switzerland., Naduvilekoot Devasia A; Division of Pediatric Neurology, University of Basel Children's Hospital, Basel, Switzerland., Zuesli S; Division of Pediatric Neurology, University of Basel Children's Hospital, Basel, Switzerland., Bernert G; Department of Pediatrics, Kaiser Franz Josef Hospital, Vienna, Austria., Laugel V; Laboratoire de Génétique Médicale, INSERM 1112, Faculté de Médecine, Strasbourg, France., Bloetzer C; Division of Pediatric Neurology, Lausanne University Hospital, Lausanne, Switzerland., Steinlin M; Division of Pediatric Neurology, University of Berne Hospital, Berne, Switzerland., Capone A; Division of Pediatric Neurology, Children's Hospital, Aarau, Switzerland., Gloor M; Department of Radiology, Division of Radiological Physics, University of Basel Hospital, Basel, Switzerland., Tobler P; Division of Pediatric Neurology, University of Basel Children's Hospital, Basel, Switzerland., Haas T; Department of Radiology, Division of Radiological Physics, University of Basel Hospital, Basel, Switzerland., Bieri O; Department of Radiology, Division of Radiological Physics, University of Basel Hospital, Basel, Switzerland., Zumbrunn T; Department of Clinical Research, Clinical Trial Unit, University of Basel Hospital, Basel, Switzerland., Fischer D; Division of Pediatric Neurology, University of Basel Children's Hospital, Basel, Switzerland; Department of Neurology, University of Basel Hospital, Basel, Switzerland; Division of Neurology, Medical University Clinic, Kantonsspital Baselland, Bruderholz, Switzerland. Electronic address: dirk.fischer@ukbb.ch., Bonati U; Division of Pediatric Neurology, University of Basel Children's Hospital, Basel, Switzerland; Department of Neurology, University of Basel Hospital, Basel, Switzerland.

Publikováno v: Neuromuscular disorders : NMD [Neuromuscul Disord] 2018 Jan; Vol. 28 (1), pp. 16-23. Date of Electronic Publication: 2017 Nov 21.