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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