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Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial.
Bertini, Enrico; Dessaud, Eric; Mercuri, Eugenio; Muntoni, Francesco; Kirschner, Janbernd; Reid, Carol; Lusakowska, Anna; Comi, Giacomo P; Cuisset, Jean-Marie; Abitbol, Jean-Louis; Scherrer, Bruno; Ducray, Patricia Sanwald; Buchbjerg, Jeppe; Vianna, Eduardo; van der Pol, W Ludo; Vuillerot, Carole; Blaettler, Thomas; Fontoura, Paulo.
Afiliação
  • Bertini E; Department of Neurosciences and Neurorehabilitation, Bambino Gesù Children's Research Hospital IRCCS, Rome, Italy. Electronic address: enricosilvio.bertini@opbg.net.
  • Dessaud E; Trophos, Marseille, France.
  • Mercuri E; Paediatric Neurology and Nemo Center, Catholic University and Policlinico Gemelli, Rome, Italy.
  • Muntoni F; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children, London, UK.
  • Kirschner J; Department of Neuropediatrics and Muscle Disorders, Medical Center-University of Freiburg, Freiburg, Germany.
  • Reid C; Biostatistics, Roche Products Limited, Welwyn Garden City, UK.
  • Lusakowska A; Department of Neurology, Medical University of Warsaw, Warsaw, Poland.
  • Comi GP; Dino Ferrari Center, Department of Pathophysiology and Transplantation, University of Milan, Neurology Unit, IRCCS Foundation Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
  • Cuisset JM; Department of Neuropediatrics, Neuromuscular Disease Reference Centre, Roger-Salengro Hospital, Regional University Teaching Hospital, Lille, France.
  • Abitbol JL; Trophos, Marseille, France.
  • Scherrer B; Bruno Scherrer Conseil, Saint-Arnoult-en-Yvelines, France.
  • Ducray PS; Roche Pharma Research and Early Development, Clinical Pharmacology, Roche Innovation Center Basel, Switzerland.
  • Buchbjerg J; Neuroscience Product Development, F Hoffmann-La Roche Ltd, Basel, Switzerland.
  • Vianna E; Neuroscience Product Development, F Hoffmann-La Roche Ltd, Basel, Switzerland.
  • van der Pol WL; Department of Neurology and Neurosurgery, Brain Center Rudolf Magnus, University Medical Center Utrecht, Utrecht, Netherlands.
  • Vuillerot C; Department of Paediatric Physical Medicine and Rehabilitation, Hôpital Femme Mère Enfant, Centre Hospitalier Universitaire de Lyon, France.
  • Blaettler T; Neuroscience Product Development, F Hoffmann-La Roche Ltd, Basel, Switzerland.
  • Fontoura P; Neuroscience Product Development, F Hoffmann-La Roche Ltd, Basel, Switzerland.
Lancet Neurol ; 16(7): 513-522, 2017 07.
Article em En | MEDLINE | ID: mdl-28460889
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Atrofias Musculares Espinais da Infância / Colestenonas / Avaliação de Resultados em Cuidados de Saúde / Fármacos Neuroprotetores Idioma: En Ano de publicação: 2017 Tipo de documento: Article
Texto completo
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XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Atrofias Musculares Espinais da Infância / Colestenonas / Avaliação de Resultados em Cuidados de Saúde / Fármacos Neuroprotetores Idioma: En Ano de publicação: 2017 Tipo de documento: Article