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Pediatric Neuroinflammatory Disease in the Era of COVID-19: Single Center Survey of the Medical and Social Impact of COVID-19 and COVID-19 Vaccines

Author:Bahri, Mara   Hurst, Lauren   Alexander, Robin   Hutchinson, Melissa   

Session Name:S34: Child Neurology and Developmental Neurology 2  

Topic:Child Neurology and Developmental Neurology  

Program Number:S34.006  

Author Institution:The Ohio State University College of Medicine, Columbus, OH  

PRAX-562 is a Well-Tolerated, Novel Persistent Sodium Channel Blocker with Broad Anticonvulsant Activity in Multiple DEE Mouse Models

Author:Anderson, Lyndsey   Jancovski, Nikola   Eckert, William   Baker, Erin   Wagley, Pravin   Maljevic, Snezana   Reid, Christopher   Patel, Manoj   Kearney, Jennifer   Kahlig, Kristopher   Petrou, Steven   

Session Name:S34: Child Neurology and Developmental Neurology 2  

Topic:Child Neurology and Developmental Neurology  

Program Number:S34.007  

Author Institution:Praxis Precision Medicines, Boston, MA  Florey Institute of Neuroscience and Mental Health, Parkville, Australia  Northwestern University Feinberg School of Medicine, Chicago, IL  University of Virginia Health System, Charlottesville, VA  

Ataluren Preserves Upper Limb Function in nmDMD Patients from Study 041, a Phase 3 Placebo-Controlled Trial, and the STRIDE Registry

Author:McDonald, Craig M.   Mercuri, Eugenio   Muntoni, Francesco   Gordish-Dressman, Heather   Morgenroth, Lauren P.   Dutra de Resende, Maria Bernadete   Zhou, Shuizhen   Neeharika, Mathukumalli Lakshmi   Haginoya, Kazuhiro   Ramos-Platt, Leigh   Williams, Paula   Penematsa, Vinay   Chou, Connie   Lin, Min   Johnson, Shelley   Koladicz, Karyn   Mastrandrea, Nicholas   Werner, Christian   Trifillis, Panayiota   

Session Name:S34: Child Neurology and Developmental Neurology 2  

Topic:Child Neurology and Developmental Neurology  

Program Number:S34.008  

Author Institution:University of California Davis School of Medicine, Davis, CA  Catholic University, Rome, Italy  UCL Institute of Child Health, London, United Kingdom  Children’s National Health System and the George Washington, Washington, DC  Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS), Pittsburgh, PA  Universidade de São Paulo, São Paulo, Brazil  Children's Hospital of Fudan University & National Children Medical Center, Shanghai, China, People's Republic of  Nizam’s Institute of Medical Sciences, Hyderabad, India  Miyagi Children'S Hospital, Sendai, Japan  University of Southern California and Children's Hospital of Los Angeles, Los Angeles, CA  PTC Therapeutics Inc., South Plainfield, NJ  PTC Therapeutics Germany GmbH, Frankfurt, Germany  

SUNFISH Parts 1 and 2: 4-year Efficacy and Safety Data of Risdiplam in Types 2 and 3 Spinal Muscular Atrophy (SMA)

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  

Evaluation of the Biodistribution, Efficacy, and Side-Effect Profile of Deflazacort, Prednisolone and Vamorolone in a DMD Mouse Model

Author:Liu, Grace   Lipari, Phil   Mollin, Anna   Jung, Stephen   Teplova, Irina   Ma, Marcia   Li, Wencheng   Ying, Lanqing   Yeh, Shirley   Narasimhan, Jana   Trifillis, Panayiota   Leonardi, Efthimia   Welch, Ellen   Voronin, Gregory   Dong, Elaine   Mastrandrea, Nicholas   Blaize, Jonathan   Weetall, Marla   

Session Name:S34: Child Neurology and Developmental Neurology 2  

Topic:Child Neurology and Developmental Neurology  

Program Number:S34.010  

Author Institution:PTC Therapeutics, Inc., South Plainfield, NJ