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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  

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Real-world Outcomes of Nusinersen or Onasemnogene Abeparvovec (OA) Monotherapy, or Switching to OA from Nusinersen in SMA Patients Aged =6 Months

Author:Dabbous, Omar   Yang, Min   Georgieva, Mihaela   Toro, Walter   LaMarca, Nicole   Patel, Anish   Anderson, Annika   Akbarnejad, Hanane   Reyna, Sandra P.   

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

Topic:Child Neurology and Developmental Neurology  

Program Number:P7.001  

Author Institution:Novartis Gene Therapies, Inc., Bannockburn, IL  Analysis Group, Inc., Boston, MA  Analysis Group, Inc., New York, NY  

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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  

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Outcomes in Patients with Spinal Muscular Atrophy and Four or More SMN2 Copies Treated with Onasemnogene Abeparvovec: Findings from RESTORE

Author:Finkel, Richard   Benguerba, Kamal   Gehani, Manish   Raju, Dheeraj   Faulkner, Eric   LaMarca, Nicole   Servais, Laurent   

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

Topic:Child Neurology and Developmental Neurology  

Program Number:P7.007  

Author Institution:St. Jude Children's Research Hospital, Memphis, TN  Novartis Gene Therapies Switzerland GmbH, Rotkreuz, Switzerland  CONEXTS-Real World Evidence, Novartis Healthcare Pvt. Ltd., Hyderabad, India  Novartis Gene Therapies, Inc., Bannockburn, IL  University of Oxford, Oxford, United Kingdom  

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Bulbar Function in Children with Two or Three SMN2 Copies Who Received Onasemnogene Abeparvovec Presymptomatically for Spinal Muscular Atrophy

Author:McGrattan, Katlyn   Shell, Richard   Hurst-Davis, Rebecca   Dunaway Young, Sally   Baranello, Giovanni   Lavrov, Arseniy   O'Brien, Eamonn   Wallach, Shiri   LaMarca, Nicole   Reyna, Sandra P.   Darras, Basil   

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

Topic:Child Neurology and Developmental Neurology  

Program Number:P7.008  

Author Institution:University of minnesota, Minneapolis, MN  Nationwide Children’s Hospital and Department of Pediatrics, The Ohio State University, Columbus, OH  Primary Children’s Hospital, Salt Lake City, UT  Stanford University School of Medicine, Stanford, CA  The Dubowitz Neuromuscular Centre, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health and NIHR Great Ormond Street Hospital Biomedical Research Centre & Great Ormond Street Hospital NHS Foundation Trust, London, United Kingdom  Novartis Gene Therapies, Inc., Cambridge, United Kingdom  Novartis, Dublin, Ireland  Novartis Gene Therapies, Inc., Bannockburn, IL  Boston Children’s Hospital, Harvard Medical School, Boston, MA  

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Displaying 1 - 5 of 6

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