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European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy.
Kirschner, Janbernd; Butoianu, Nina; Goemans, Nathalie; Haberlova, Jana; Kostera-Pruszczyk, Anna; Mercuri, Eugenio; van der Pol, W Ludo; Quijano-Roy, Susana; Sejersen, Thomas; Tizzano, Eduardo F; Ziegler, Andreas; Servais, Laurent; Muntoni, Francesco.
Afiliação
  • Kirschner J; Department of Neuropediatrics, University Hospital Bonn, Bonn, Germany. Electronic address: Janbernd.kirschner@ukbonn.de.
  • Butoianu N; Pediatric Neurology Clinic, "Prof. Dr. Al. Obregia" Hospital, Bucharest, Faculty of Medicine and Pharmacy "Carol Davila", Bucharest, Romania. Electronic address: nbutoianu91@gmail.com.
  • Goemans N; Dept of Pediatric Neurology, University Hospitals Leuven, Belgium. Electronic address: nathalie.goemans@uzleuven.be.
  • Haberlova J; Dept of Pediatric Neurology, Motol University Hospital, Prague, Czech Republic. Electronic address: Jana.Haberlova@fnmotol.cz.
  • Kostera-Pruszczyk A; Department of Neurology, Medical University of Warsaw, Poland. Electronic address: anna.kostera-pruszczyk@wum.edu.pl.
  • Mercuri E; Pediatric Neurology, Università Cattolica del Sacro Cuore, Rome, Italy; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy. Electronic address: eumercuri@gmail.com.
  • van der Pol WL; Department of Neurology, UMC Utrecht Brain Center, University Medical Center Utrecht, Utrecht University, the Netherlands. Electronic address: W.L.vanderPol@umcutrecht.nl.
  • Quijano-Roy S; Neuromuscular Unit, Child Neurology and ICU Department, Raymond Poincaré University Hospital (UVSQ), APHP Paris Saclay, Garches, France. Electronic address: susana.quijano-roy@aphp.fr.
  • Sejersen T; Department of Womens and Childrens Health, Karolinska Institutet, Stockholm, Sweden. Electronic address: thomas.sejersen@ki.se.
  • Tizzano EF; Department of Clinical and Molecular Genetics, Medicine Genetics Group, University Hospital Vall d'Hebron, Barcelona, Spain. Electronic address: etizzano@vhebron.net.
  • Ziegler A; Department of Neuropediatrics and Metabolic Medicine, Centre for Childhood and Adolescent Medicine, University Hospital Heidelberg, Germany. Electronic address: andreas.ziegler@med.uni-heidelberg.de.
  • Servais L; University of Liège, Neuromuscular Reference Center Disease, Department of Pediatrics, Liege, Belgium; MDUK Neuromuscular Center, Department of Pediatrics, University of Oxford, UK. Electronic address: laurent.servais@paediatrics.ox.ac.uk.
  • Muntoni F; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, and NIHR Biomedical Research Centre, Great Ormond Street Hospital for Children, London, UK. Electronic address: f.muntoni@ucl.ac.uk.
Eur J Paediatr Neurol ; 28: 38-43, 2020 Sep.
Article em En | MEDLINE | ID: mdl-32763124
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Produtos Biológicos / Proteínas Recombinantes de Fusão / Atrofia Muscular Espinal / Terapia Genética Tipo de estudo: Guideline Limite: Humans / Infant Idioma: En Ano de publicação: 2020 Tipo de documento: Article
Texto completo
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Produtos Biológicos / Proteínas Recombinantes de Fusão / Atrofia Muscular Espinal / Terapia Genética Tipo de estudo: Guideline Limite: Humans / Infant Idioma: En Ano de publicação: 2020 Tipo de documento: Article