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Displaying 41 - 45 of 116

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Baseline Characteristics and Interim Safety in RESPOND: A Phase 4 Study in Children with Spinal Muscular Atrophy (SMA) Treated With Nusinersen After Onasemnogene Abeparvovec

Author:Kuntz, Nancy   Parsons, Julie   Brandsema, John F.   Proud, Crystal   Finkel, Richard   Swoboda, Kathryn   Masson, Ricardo   Liu, Yingying   Makepeace, Corinne   Paradis, Angela   Berger, Zdenek   Somera-Molina, Kathleen   

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

Topic:Child Neurology and Developmental Neurology  

Program Number:P7.003  

Author Institution:Ann & Robert H Lurie Children'S Hospital of Chicago, Chicago, IL  Children's Hospital Colorado, Aurora, CO  Children’s Hospital of Philadelphia, Philadelphia, PA  Children's Hospital of The King's Daughters, Norfolk, VA  St. Jude Children's Research Hospital, Memphis, TN  Massachusetts General Hospital, Boston, MA  Fondazione IRCCS Istituto Neurologico Carlo Besta , Milano, Italy  Biogen, Cambridge, MA  Biogen, Maidenhead, United Kingdom  

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JEWELFISH: 24-month Safety, Pharmacodynamic and Exploratory Efficacy Data in Non-Treatment-Naïve Patients with Spinal Muscular Atrophy (SMA) Receiving Treatment with Risdiplam

Author:Chiriboga, Claudia   Bruno, Claudio   Duong, Tina   Fischer, Dirk   Kirschner, Janbernd   Scoto, Mariacristina   Mercuri, Eugenio   Gerber, Marianne   Gorni, Ksenija   Kletzl, Heidemarie   Carruthers, Imogen   Martin, Carmen   Gidaro, Teresa   Muntoni, Francesco   

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

Topic:Child Neurology and Developmental Neurology  

Program Number:P7.004  

Author Institution:Columbia Irving Medical Center, New York, NY  Istituto Giannina Gaslini, Genoa, Italy  Stanford University, Palo Alto, CA  University Children’s Hospital Basel, Basel, Switzerland  University of Freiburg, Freiburg, Germany  Great Ormond Street Institute of Child Health University College London, & Great Ormond Street Hospital Trust, London, United Kingdom  Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy  F. Hoffmann-La Roche Ltd, Basel, Switzerland  Roche Innovation Center Basel, Basel, Switzerland  Roche Products Ltd, Welwyn Garden City, United Kingdom  Hoffmann-La Roche Ltd, Basel, Switzerland  

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Feasibility of a Home-Based Exergame Therapy for Youth with Spinal Muscular Atrophy

Author:Oskoui, Maryam   Selby, Kathy   Herzig, David   Cardiff, Katrina   Cushen, Niamh   Defosses, Yan   Gonorazky, Hernan   Gottowik, Juergen   Haldenby, Renee   Jurisic, Ivan   Karthigesu, Shaainthabie   MacIntyre, Leigh   MacKenzie, Alex   Mah, Jean   McCullough, James   Ng, Pamela   Opalka, Slawomir   Openchowski, Michal   Petkun, Svetlana   Potter, Beth   Sheriko, Jordan   Smith, Maureen   Strahm, Martin   Turgeon Desilets, Sarah   Woof, Angelina   Chen, Xing   

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

Topic:Child Neurology and Developmental Neurology  

Program Number:P7.005  

Author Institution:McGill University , Montreal, Quebec, QC, Canada  

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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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Displaying 41 - 45 of 116

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