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Safety and efficacy of nusinersen in spinal muscular atrophy: The EMBRACE study.
Acsadi, Gyula; Crawford, Thomas O; Müller-Felber, Wolfgang; Shieh, Perry B; Richardson, Randal; Natarajan, Niranjana; Castro, Diana; Ramirez-Schrempp, Daniela; Gambino, Giulia; Sun, Peng; Farwell, Wildon.
Afiliação
  • Acsadi G; Division of Pediatric Neurology, Connecticut Children's Medical Center, University of Connecticut School of Medicine, Farmington, Connecticut, USA.
  • Crawford TO; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
  • Müller-Felber W; Department of Neuropediatrics, Developmental Neurology and Social Pediatrics, LMU Campus Innenstadt, University of Munich, Munich, Germany.
  • Shieh PB; Department of Neurology, UCLA Clinical and Translational Research Center, Los Angeles, California, USA.
  • Richardson R; Pediatric Neurology, Gillette Children's Specialty Healthcare, St Paul, Minnesota, USA.
  • Natarajan N; Department of Neurology, Seattle Children's Research Institute, Seattle, Washington, USA.
  • Castro D; Department of Pediatrics, Neurology and Neurotherapeutics, UT Southwestern Medical Center, Dallas, Texas, USA.
  • Ramirez-Schrempp D; Biogen, Cambridge, Massachusetts, USA.
  • Gambino G; Biogen, Maidenhead, UK.
  • Sun P; Biogen, Cambridge, Massachusetts, USA.
  • Farwell W; Biogen, Cambridge, Massachusetts, USA.
Muscle Nerve ; 63(5): 668-677, 2021 05.
Article em En | MEDLINE | ID: mdl-33501671

Texto completo: 1 Bases de dados: MEDLINE Assunto principal: Oligonucleotídeos / Atrofias Musculares Espinais da Infância Tipo de estudo: Clinical_trials Limite: Child, preschool / Female / Humans / Infant / Male Idioma: En Revista: Muscle Nerve Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Estados Unidos

Texto completo: 1 Bases de dados: MEDLINE Assunto principal: Oligonucleotídeos / Atrofias Musculares Espinais da Infância Tipo de estudo: Clinical_trials Limite: Child, preschool / Female / Humans / Infant / Male Idioma: En Revista: Muscle Nerve Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Estados Unidos