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Intravenous and Intrathecal Onasemnogene Abeparvovec Gene Therapy in Symptomatic and Presymptomatic Spinal Muscular Atrophy: Long-Term Follow-Up Study

Author:Darras, Basil   Mercuri, Eugenio   Strauss, Kevin   Day, John   Chien, Yin-Hsiu   Masson, Ricardo   Wigderson, Melissa   Alecu, Iulian   Ballarini, Nicolas   Mehl, Lesa   Marra, Jonathan   Connolly, Anne   

Session Name:S34: Child Neurology and Developmental Neurology 2  

Topic:Child Neurology and Developmental Neurology  

Program Number:S34.002  

Author Institution:Boston Children’s Hospital, Harvard Medical School, Boston, MA  Department of Paediatric Neurology and Nemo Clinical Centre, Catholic University, Rome, Italy  Clinic for Special Children, Strasburg, PA  Stanford University School of Medicine, Palo Alto, CA  National Taiwan University Hospital, Taipei City, Taiwan  Fondazione IRCCS Istituto Neurologico Carlo Besta, Milano, Italy  Novartis Gene Therapies, Inc., Bannockburn, IL  Novartis Pharmaceuticals, Basel, Switzerland  Novartis Institutes for BioMedical Research, Cambridge, MA  Novartis Pharmaceuticals, East Hanover, NJ  Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH  

Long-Term Follow-Up of Onasemnogene Abeparvovec Gene Therapy in Patients with Spinal Muscular Atrophy Type 1

Author:Mendell, Jerry   Wigderson, Melissa   Alecu, Iulian   Yang, Lina   Mehl, Lesa   Connolly, Anne   

Session Name:P7: Child Neurology and Developmental Neurology: Spinomuscular Atrophy/Muscular Dystrophies  

Topic:Child Neurology and Developmental Neurology  

Program Number:P7.006  

Author Institution:Center for Gene Therapy, Nationwide Children’s Hospital, Columbus, OH  Novartis Gene Therapies, Inc., Bannockburn, IL  Novartis Pharmaceuticals, Basel, Switzerland  Novartis Gene Therapies, Inc., East Hanover, NJ  Novartis Institutes for BioMedical Research, Cambridge, MA  Nationwide Children's Hospital, Columbus, OH