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Continued benefit of nusinersen initiated in the presymptomatic stage of spinal muscular atrophy: 5-year update of the NURTURE study.
Crawford, Thomas O; Swoboda, Kathryn J; De Vivo, Darryl C; Bertini, Enrico; Hwu, Wuh-Liang; Finkel, Richard S; Kirschner, Janbernd; Kuntz, Nancy L; Nazario, Aledie Navas; Parsons, Julie A; Pechmann, Astrid; Ryan, Monique M; Butterfield, Russell J; Topaloglu, Haluk; Ben-Omran, Tawfeg; Sansone, Valeria A; Jong, Yuh-Jyh; Shu, Francy; Zhu, Cong; Raynaud, Stephanie; Lago, Tiffany R; Paradis, Angela D; Foster, Richard; Chin, Russell; Berger, Zdenek.
Afiliação
  • Crawford TO; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.
  • Swoboda KJ; Department of Neurology, Center for Genomic Medicine, Massachusetts General Hospital, Boston, Massachusetts, USA.
  • De Vivo DC; Departments of Neurology and Pediatrics, Columbia University Irving Medical Center, New York, New York, USA.
  • Bertini E; Unit of Neuromuscular and Neurodegenerative Disorders, Post-Graduate Bambino Gesù Children's Research Hospital, IRCCS, Rome, Italy.
  • Hwu WL; Departments of Medical Genetics and Pediatrics, National Taiwan University Hospital, Taipei, Taiwan.
  • Finkel RS; Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
  • Kirschner J; Division of Neurology, Nemours Children's Hospital, Orlando, Florida, USA.
  • Kuntz NL; Department of Neuropediatrics and Muscle Disorders, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
  • Nazario AN; Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.
  • Parsons JA; Division of Pulmonary Medicine, Department of Pediatrics, Nemours Children's Hospital, Orlando, Florida, USA.
  • Pechmann A; Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, Colorado, USA.
  • Ryan MM; Department of Neuropediatrics and Muscle Disorders, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
  • Butterfield RJ; Murdoch Children's Research Institute and University of Melbourne, Melbourne, Australia.
  • Topaloglu H; Department of Pediatrics and Neurology, University of Utah, Salt Lake City, Utah, USA.
  • Ben-Omran T; Department of Pediatric Neurology, Hacettepe University, Ankara, Turkey.
  • Sansone VA; Division of Genetic and Genomic Medicine, Sidra Medicine, Doha, Qatar.
  • Jong YJ; Medical Genetics Department, Hamad Medical Corporation, Doha, Qatar.
  • Shu F; The NeMo Clinical Center, Neurorehabilitation Unit, University of Milan, Milan, Italy.
  • Zhu C; Department of Biomedical Sciences for Health, Universitàdegli Studi di Milano, Milan, Italy.
  • Raynaud S; Graduate Institute of Clinical Medicine, College of Medicine, Kaohsiung Medical University Hospital, Kaohsiung Medical University, Kaohsiung, Taiwan.
  • Lago TR; Translational Research Center of Neuromuscular Diseases, and Departments of Pediatrics and Laboratory Medicine, Kaohsiung Medical University Hospital, Kaohsiung Medical University, Kaohsiung, Taiwan.
  • Paradis AD; Department of Neurology, David Geffen School of Medicine at UCLA, Los Angeles, California, USA.
  • Foster R; Biogen, Cambridge, Massachusetts, USA.
  • Chin R; Biogen, Cambridge, Massachusetts, USA.
  • Berger Z; Biogen, Cambridge, Massachusetts, USA.
Muscle Nerve ; 68(2): 157-170, 2023 08.
Article em En | MEDLINE | ID: mdl-37409780
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Atrofia Muscular Espinal / Atrofias Musculares Espinais da Infância Limite: Child / Humans Idioma: En Revista: Muscle Nerve Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Estados Unidos
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Atrofia Muscular Espinal / Atrofias Musculares Espinais da Infância Limite: Child / Humans Idioma: En Revista: Muscle Nerve Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Estados Unidos