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Author:Servais, Laurent Oskoui, Maryam Day, John Deconinck, Nicholas Mazzone, Elena Nascimento, Andrés Saito, Kyoko Vuillerot, Carole Baranello, Giovanni Boespflug-Tanguy, Odile Goemans, Nathalie Kirschner, Janbernd Kostera-Pruszczyk, Anna Braid, Jessica Papp, Gergely Gorni, Ksenija Martin, Carmen Scalco, Renata Yeung, Wai Yin Mercuri, Eugenio
Session Name:S34: Child Neurology and Developmental Neurology 2
Topic:Child Neurology and Developmental Neurology
Program Number:S34.009
Author Institution:IMotion Institut de Myologie AP-HP, Hôpital Armand Trousseau, Paris, France MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, UK Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, Department of Pediatrics, University Hospital Liège & University of Liège, Liège, Belgium, Oxford, United Kingdom
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
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
Author:Darras, Basil Baranello, Giovanni Boespflug-Tanguy, Odile Day, John Deconinck, Nicholas Klein, Andrea Masson, Ricardo Mazurkiewicz-Beldzinska, Maria Mercuri, Eugenio Rose, Kristy Vlodavets, Dmitry Xiong, Hui Zanoteli, Edmar El-Khairi, Muna Gerber, Marianne Gorni, Ksenija Kletzl, Heidemarie Palfreeman, Laura Dodman, Angela Servais, Laurent
Program Number:P7.009
Author Institution:Department of Neurology, Boston Children’s Hospital, Harvard Medical School, Boston, MA, USA, Boston, MA Dubowitz Neuromuscular Centre, University College London, London, UK Developmental Neurology, Istituto Neurologico C Besta, Milan, Italy, London, United Kingdom IMotion Institut de Myologie AP-HP, Hôpital Armand Trousseau, Paris, France Université Paris Cité, UMR 1141, NeuroDiderot, Paris, France, Paris, France Stanford University, Palo Alto, CA, USA, Palo Alto, CA Neuromuscular Center, UZ Gent, Ghent, Belgium Centre de Référence des Maladies Neuromusculaires, Children’s University Hospital, ULB, Brussels, Belgium, Ghent, Belgium Paediatric Neurology, Uni. Children’s Hospital Basel, Basel, Switzerland Paediatric Neurology, Inselspital, University of Bern, Bern, Switzerland, Basel, Switzerland Developmental Neurology, Istituto Neurologico C Besta, Milan, Italy, Milan, Italy Developmental Neurology, Medical University of Gdansk, Gdansk, Poland, Gdansk, Poland Pediatric Neurology Institute, Catholic University, Rome, Italy, Rome, Italy Paediatric Gait Analysis Service of New South Wales, The Children’s Hospital at Westmead, Sydney, Australia, Sydney, Australia Children Neuromuscular Center, Pirogov Medical University, Moscow, Russia, Moscow, Russian Federation Department of Pediatrics, Peking University First Hospital, Beijing, China, Beijing, China, People's Republic of Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo, Brazil, Sao Paulo, Brazil Roche Products Ltd, Welwyn Garden City, UK, Welwyn Garden City, United Kingdom Pharma Development, Safety, F. Hoffmann-La Roche Ltd, Basel, Switzerland, Basel, Switzerland PDMA Neuro/Rare Disease, F. Hoffmann-La Roche Ltd, Basel, Switzerland, Basel, Switzerland Pharmaceutical Research, Roche Innovation Center Basel, Basel, Switzerland, Basel, Switzerland Pharma Development Neuro, F. Hoffmann-La Roche Ltd, Basel, Switzerland, Basel, Switzerland MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, UK Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, Department of Pediatrics, University of Liège, Liège, Belgium, Oxford, United Kingdom
Author:Servais, Laurent Benguerba, Kamal De Vivo, Darryl C. Kirschner, Jan Muntoni, Francesco Proud, Crystal Tizzano, Eduardo Quijano-Roy, Susana Desguerre, Isabelle Saito, Kyoko Raju, Dheeraj LaMarca, Nicole Sun, Rui Anderson, Fred Faulkner, Eric Finkel, Richard
Program Number:P7.010
Author Institution:University of Oxford, Oxford, United Kingdom Novartis Gene Therapies Switzerland GmbH, Rotkreuz, Switzerland Columbia University Irving Medical Center, New York, NY Clinic for Neuropediatrics and Muscle Disease, University Medical Center Freiburg, Freiburg, Germany The Dubowitz Neuromuscular Centre, University College London, Great Ormond Street Institute of Child Health & Great Ormond Street Hospital and National Institute of Health Research, Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom Children’s Hospital of the King’s Daughters, Norfolk, VA Hospital Valle Hebron, Barcelona, Spain Garches Neuromuscular Reference Center (GNMH), APHP Raymond Poincare University Hospital (UVSQ Paris Saclay), Garches, France Hôpital Necker Enfants Malades, APHP, Paris, France Institute of Medical Genetics, Tokyo Women’s Medical University, Tokyo, Japan Novartis Gene Therapies, Inc., Bannockburn, IL Center for Outcomes Research, University of Massachusetts Medical School, Worcester, MA St. Jude Children's Research Hospital, Memphis, TN