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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