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Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial.
Strauss, Kevin A; Farrar, Michelle A; Muntoni, Francesco; Saito, Kayoko; Mendell, Jerry R; Servais, Laurent; McMillan, Hugh J; Finkel, Richard S; Swoboda, Kathryn J; Kwon, Jennifer M; Zaidman, Craig M; Chiriboga, Claudia A; Iannaccone, Susan T; Krueger, Jena M; Parsons, Julie A; Shieh, Perry B; Kavanagh, Sarah; Wigderson, Melissa; Tauscher-Wisniewski, Sitra; McGill, Bryan E; Macek, Thomas A.
Afiliação
  • Strauss KA; Clinic for Special Children, Strasburg, PA, USA. kstrauss@clinicforspecialchildren.org.
  • Farrar MA; Penn Medicine-Lancaster General Hospital, Lancaster, PA, USA. kstrauss@clinicforspecialchildren.org.
  • Muntoni F; Departments of Pediatrics and Molecular, Cell & Cancer Biology, University of Massachusetts School of Medicine, Worcester, MA, USA. kstrauss@clinicforspecialchildren.org.
  • Saito K; Department of Neurology, Sydney Children's Hospital Network, Sydney, NSW, Australia.
  • Mendell JR; School of Clinical Medicine, UNSW Medicine and Health, UNSW Sydney, Sydney, NSW, Australia.
  • Servais L; The Dubowitz Neuromuscular Centre, University College London, Great Ormond Street Institute of Child Health & Great Ormond Street Hospital, London, UK.
  • McMillan HJ; National Institute of Health Research, Great Ormond Street Hospital Biomedical Research Centre, London, UK.
  • Finkel RS; Institute of Medical Genetics, Tokyo Women's Medical University, Tokyo, Japan.
  • Swoboda KJ; Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.
  • Kwon JM; Department of Pediatrics and Department of Neurology, The Ohio State University, Columbus, OH, USA.
  • Zaidman CM; Department of Paediatrics, MDUK Oxford Neuromuscular Centre, Oxford, UK.
  • Chiriboga CA; Neuromuscular Reference Center, Department of Pediatrics, CHU & University of Liège, Liège, Belgium.
  • Iannaccone ST; Department of Pediatrics, Neurology & Neurosurgery, Montreal Children's Hospital, McGill University, Montreal, QC, Canada.
  • Krueger JM; Department of Pediatrics, Nemours Children's Hospital, Orlando, FL, USA.
  • Parsons JA; Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, TN, USA.
  • Shieh PB; Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.
  • Kavanagh S; Department of Neurology, University of Wisconsin School of Medicine and Public Health, Madison, WI, USA.
  • Wigderson M; Washington University School of Medicine, St. Louis, MO, USA.
  • Tauscher-Wisniewski S; Division of Pediatric Neurology, Columbia University Medical Center, New York, NY, USA.
  • McGill BE; Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, USA.
  • Macek TA; Department of Neurology, Helen DeVos Children's Hospital, Grand Rapids, MI, USA.
Nat Med ; 28(7): 1390-1397, 2022 07.
Article em En | MEDLINE | ID: mdl-35715567
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Atrofia Muscular Espinal / Atrofias Musculares Espinais da Infância Tipo de estudo: Clinical_trials / Etiology_studies / Risk_factors_studies Limite: Child / Humans / Infant Idioma: En Revista: Nat Med Ano de publicação: 2022 Tipo de documento: Article
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 Tipo de estudo: Clinical_trials / Etiology_studies / Risk_factors_studies Limite: Child / Humans / Infant Idioma: En Revista: Nat Med Ano de publicação: 2022 Tipo de documento: Article