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RAINBOWFISH: Primary Efficacy and Safety Data in Risdiplam-treated Infants with Presymptomatic Spinal Muscular Atrophy (SMA)

Author:Servais, Laurent   Farrar, Michelle   Finkel, Richard   Vlodavets, Dmitry   Zanoteli, Edmar   Al-Muhaizea, Mohammad   Araújo, Alexandra Prufer de Queiroz Campos   Nelson, Leslie   Jaber, Birgit   Gorni, Ksenija   Kletzl, Heidemarie   Palfreeman, Laura   Gaki, Eleni   Rabbia, Michael   Summers, Dave   Fontoura, Paulo   Bertini, Enrico   

Session Name:S37: Emerging Therapies in Child Neurology  

Topic:Child Neurology and Developmental Neurology  

Program Number:S37.006  

Author Institution:University of Oxford, Oxford, United Kingdom  UNSW Sydney, Sydney, Australia  St. Jude Children's Research Hospital, Memphis, TN  Veltischev Clinical Pediatric Research Institute of Pirogov Russian National Research Medical University, Moscow, Russian Federation  Universidade de São Paulo, Sao Paulo, Brazil  King Faisal Specialist Hospital & Research Center-Riyadh, Riyadh, Saudi Arabia  Federal University of Rio de Janeiro, Rio de Janeiro, Brazil  University of Texas Southwestern Medical Center, Dallas, TX  Pharma Development, Safety, F. Hoffmann-La Roche Ltd, Basel, Switzerland  F. Hoffmann-La Roche Ltd, Basel, Switzerland  Roche Innovation Center Basel, Basel, Switzerland  Roche Products Ltd, Welwyn Garden City, United Kingdom  Genentech, Inc., South San Francisco, CA  Bambino Gesù Children’s Research Hospital IRCCS, Rome, Italy  

Delphi Consensus Panel on Assessing Clinically Meaningful Treatment Outcomes in Adults Living with Spinal Muscular Atrophy (SMA)

Author:Sully, Kate   Chiriboga, Claudia   Duong, Tina   Erbas, Yasemin   Glascock, Jacqueline   Gusset, Nicole   Muni-Lofra, Robert   O'Connell, Colleen   Vázquez-Costa, Juan F   Walter, Maggie C   Guittari, Carol Jean   Cochrane, James   Townson, Louisa D   Kufakwaro, Jason   Marciniak, Anne   Riley, Danielle   Nam, Julian   

Session Name:P2: Neuromuscular and Clinical Neurophysiology (EMG): Spinal Muscular Atrophy and Hereditary Neuropathies  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:P2.007  

Author Institution:Roche Products Ltd, Welwyn Garden City, United Kingdom  Columbia University Irving Medical Center, New York, NY  Stanford University, San Francisco, CA  SMA Europe, Freiburg, Germany  Cure SMA, Elk Grove Village, IL  Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom  Stan Cassidy Centre for Rehabilitation, Fredericton, NB, Canada  Hospital Universitari i Politècnic La Fe, Valencia, Spain  LMU Munich, Munich, Germany  Genentech, Inc., South San Francisco, CA  Adelphi Values PROVE™, Bollington, United Kingdom  F. Hoffmann-La Roche Ltd, Basel, Switzerland