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FIREFISH Parts 1 and 2: 36-month safety and efficacy of risdiplam in Type 1 spinal muscular atrophy (SMA)

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   

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

Topic:Child Neurology and Developmental Neurology  

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  

Treatments and Outcomes for Patients with Spinal Muscular Atrophy Type 2: Findings from RESTORE Registry

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   

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

Topic:Child Neurology and Developmental Neurology  

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