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Efficacy and Safety of Zilganersen, an Investigational RNA-targeted Antisense Therapy, in People Living with Alexander Disease: Results from a Pivotal Study

Author:Waldman, Amy   Lynch, David   Tonduti, Davide   Van Der Knaap, Marjo   Bernard, Geneviève   Eichler, Florian   Sampson, Jacinda   Zerem, Ayelet   Chataway, Jeremy   Cunningham, Chloe   Bertini, Enrico   Hacohen, Yael   Saito, Takashi   Keller, Stephanie   Vaia, Ylenia   Kim, Nee Na   Nagy, Amanda   Van Haren, Keith   Van den Berg, Elske   Chen, Chen   Ruiz, Norma   Collins, Abigail   Smith, Anne   

Session Name:PL5: Clinical Trials Plenary Session  

Topic:Child Neurology and Developmental Neurology  

Program Number:PL5.003  

Author Institution:Division of Neurology, Children’s Hospital of Philadelphia, and Departments of Neurology and Pediatrics, Perelman School of Medicine at the University of Pennsylvania, PA  Queen Square Multiple Sclerosis Centre, Department of Neuroinflammation, UCL Queen Square Institute of Neurology, Faculty of Brain Sciences, University College London  National Institute for Health Research, University College London Hospitals, Biomedical Research Centre  Inherited White Matter Disorders Highly Specialised Service, National Hospital for Neurology and Neurosurgery, University College London Hospitals NHS Foundation Trust, United Kingdom  Unit of Pediatric Neurology, COALA (Center for Diagnosis and Treatment of Leukodystrophies), V. Buzzi Children's Hospital, University of Milan, MILANO, Italy  Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam, Netherlands  Department of Neurology and Neurosurgery, Pediatrics and Human Genetics, McGill University, QC, Canada  Department of Neurology, Massachusetts General Hospital, Harvard Medical School, and the Center for Genomic Medicine, Massachusetts General Hospital, MA  Department of Neurology and Neurological Sciences, Stanford Neuroscience Health Center, Palo Alto, CA  Dana-Dwek Children’s Hospital, Tel Aviv Sourasky Medical Center, Tel Aviv University, Tel Aviv, Israel  Murdoch Children’s Research Institute, Parkville, Australia  Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children’s Hospital, IRCCS, Rome, Italy  Department of Pediatric Neurology, Great Ormond Street Hospital for Children, United Kingdom  Department of Child Neurology , National Center of Neurology and Psychiatry, Kodaira, Japan  Department of Pediatrics, Division of Pediatric Neurology, Emory University School of Medicine, GA  Unit of Pediatric Neurology, COALA (Center for Diagnosis and Treatment of Leukodystrophies), V. Buzzi Children's Hospital, University of Milan, Milan, Italy  Department of Neurology, Great Ormond Street Hospital for Children, United Kingdom  Department of Neurology, Massachusetts General Hospital, Harvard Medical School, and the Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA  Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Netherlands  Ionis, CA  Ionis, Carlsbad, CA  

Efficacy and Safety of Tolebrutinib Versus Placebo in Primary Progressive Multiple Sclerosis: Results from the Phase Three PERSEUS Trial

Author:Fox, Robert   Bar-Or, Amit   Vermersch, Patrick   Oreja Guevara, Celia   Traboulsee, Anthony   Giovannoni, Gavin   Syed, Sana   Sun, Furong   Salloum, Naji   Turner, Timothy   Wallstroem, Erik   Reich, Daniel   

Session Name:PL6: Contemporary Clinical Issues Plenary Session  

Topic:Multiple Sclerosis  

Program Number:PL6.003  

Author Institution:Cleveland Clinic, Cleveland, OH  University of Pennsylvania, Philadelphia, PA  University of Lille, Lille, France  Instituto de Investigación Sanitaria del Hospital Clínico San Carlos, Madrid, Spain  University of British Columbia, Vancouver, BC, Canada  Queen Mary University London, London, United Kingdom  Sanofi , Cambridge, MA  Sanofi, Morristown, NJ  Sanofi, CHILLY-MAZARIN, France  Sanofi, Cambridge, MA  Translational Neuroradiology Section, National Institutes of Health, Bethesda, MD  

Immunosuppressive Therapies in Down Syndrome Regression Disorder: A Prospective Observational Cohort Study

Author:Santoro, Jonathan   Kazerooni, Lilia   Lucas, Maeve   Yousuf, Mariam   Otey, Samuel   Nguyen, Lina   Anadani, Nidhiben   Manning, Melanie   Rachubinski, Angela   Kammeyer, Ryan   Spinazzi, Noemi   Franklin, Catherine   Quinn, Eileen   Espinosa, Joaquin   Rafii, Michael   

Session Name:N2: Neuroscience in the Clinic: Neurological Care of Individuals with Down Syndrome Across the Lifespan  

Topic:Child Neurology and Developmental Neurology  

Program Number:N2.002  

Author Institution:Department of Neurology, Children's Hospital Los Angeles, Los Angeles, CA  Children's Hospital Los Angeles, Los Angeles, CA  Children's Hospital Los Angeles, CA  University Of Oklahoma Health Science Center, Oklahoma City, OK  Stanford School of Medicine, Palo Alto, CA  Linda Crnic Institute for Down Syndrome, Aurora, CO  Childrens Hospital Colorado, Aurora, CO  University of California San Francisco, Oakland, CA  The University of Queensland, South Brisbane, Australia  University of Toledo, Toledo, OH  USC Alzheimer'S Therapeutic Research Institute, San Diego, CA  

Efficacy and Safety of Fenebrutinib vs Teriflunomide in Relapsing Multiple Sclerosis: Results of the FENhance 1 and 2 Studies

Author:Oh, Jiwon   Bar-Or, Amit   Giovannoni, Gavin   Sormani, Maria Pia   Weber, Martin S.   Stoll, Sharon   Nicholas, Jacqueline A.   von Büdingen, H.-Christian   Decker-Palmer, Marquita   Loukitcheva, Anastasia   Triyatni, Miriam   Hu, Nan   Ratchford, John N.   Napieralski, Julie   Goodyear, Alexandra   Kappos, Ludwig   Hauser, Stephen   

Session Name:LS2: Late-breaking Science 2  

Topic:Multiple Sclerosis  

Program Number:LS2.005  

Author Institution:St. Michael's Hospital, University of Toronto, Toronto, ON, Canada  Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA  Queen Mary University of London, London, United Kingdom  University of Genoa, Genoa, Italy  Institute of Neuropathology and Department of Neurology, University Medical Center and Fraunhofer Institute for Translational Medicine and Pharmacology , Göttingen, Germany  Advocare Stoll Medical Group, Philadelphia, PA  OhioHealth Multiple Sclerosis Centre, Riverside Methodist Hospital, Columbus, OH  F. Hoffmann-La Roche Ltd, Basel, Switzerland  Genentech, Inc., South San Francisco, CA  University Hospital and University Basel, Research Center for Clinical Neuroimmunology and Neuroscience (RC2NB), Basel, Switzerland  University of California San Francisco, San Francisco, CA  

Safety and Efficacy of Riliprubart, an Activated C1s-complement Inhibitor in CIDP: 76-week Phase Two Trial Results

Author:Querol, Luis   Lewis, Richard A.   Hartung, Hans-Peter   van Doorn, Pieter A.   Lin, Jie   Dionne, Annie   Attarian, Shahram   Wallstroem, Erik   Auwarter, Kristen   Lu, Yi   Alonso-Alonso, Miguel   Atassi, Nazem   Hughes, Richard A. C.   

Session Name:S2: Updates on Motor Neuron and Peripheral Nerve Disorders  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S2.008  

Author Institution:Neuromuscular Diseases Unit, Department of Neurology, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain  Centro para la Investigación Biomédica en Red en Enfermedades Raras (CIBERER), Madrid, Spain, Spain  Department of Neurology, Cedars Sinai Medical Center, Los Angeles, CA, USA, CA  Department of Neurology, Faculty of Medicine, Heinrich-Heine-University, Düsseldorf, Germany  Brain and Mind Center, University of Sydney, Sydney, NSW, Australia  Department of Neurology, Medical University of Vienna, Vienna, Austria  Department of Neurology, Palacky University Olomouc, Olomouc, The Czech Republic, Dusseldorf, Germany  Erasmus MC, University Medical Center, Rotterdam, The Netherlands, Netherlands  Department of Neurology and Rare Disease Center, Huashan Hospital, Fudan University, Shanghai, China, Shanghai, China, People's Republic of  CHU de Quebec Universite Laval, Quebec, Canada, QC, Canada  Neuromuscular Disease and ALS Reference Center, Timone University Hospital, Aix-Marseille University, CHU Timone, Marseille Cedex 05, France, France  Sanofi R&D, Neurology Development, Cambridge, MA, USA, Cambridge, MA  Sanofi, USA, MA  Sanofi R&D, Biostatistics and Programming, Cambridge, MA, USA, CA  Sanofi R&D, Neurology Development, Cambridge, MA, USA, MA  UCL Queen Square Institute of Neurology, University College London, London, UK, United Kingdom