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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  

A Prospective Longitudinal Observational Study in Myotonic Dystrophy Type One: From Clinical Outcomes to Trial Design

Author:Sansone, Valeria   Ferrari Aggradi, Carola   Lizio, Andrea   Nani, Michela   Dekdebrun, Jeanne   Eichinger, Katy   Gagnon, Cynthia   Subramony, S   Roxburgh, Richard   Hamel, Johanna   Statland, Jeffrey   Mul, Karlien   van Engelen, Baziel   Elsheikh, Bakri   Arnold, William   Turner, Chris   Schoser, Benedikt   Ragole, Thomas   Matthews, Emma   Sampson, Jacinda   Takahashi, Massanori   Wicklund, Matthew   Swenson, Andrea   Laverty, Chamindra   Shieh, Perry   Greene, Ericka   Hung, Man   Langeslay, Ruby   Raymond, Jennifer   Thornton, Charles   Johnson, Nicholas   

Session Name:P8: Neuromuscular and Clinical Neurophysiology (EMG): Muscle Disorders  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:P8.003  

Author Institution:The NeMO Clinical Center - Neurorehabilitation Unit, University of Milan, Milan, Italy  University of Rochester, NY  University of Rochester, Rochester, NY  Sherbrooke University, QC, Canada  University of Florida, Gainesville, FL  Auckland Hospital, Auckland, New Zealand  University of Rochester, Neurology, Rochester, NY  University of Kansas Medical Center, Kansas City, KS  Radboud University, Nijmegen, Netherlands  Radboud University Nijmegen Medical Centre, Nijmegen, Netherlands  The Ohio State University Wexner Medical Center, Columbus, OH  University of Missouri, Columbus, OH  University College London, United Kingdom  Friedrich-Baur Institut, Munich, Germany  University of Colorado Department of Neurology, Aurora, CO  Atkinson-Morley Neuromuscular Centre, London, United Kingdom  Stanford University, PALO ALTO, CA  Clinical Neurophysiology, Osaka University, Suita, Japan  UT Health San Antonio, San Antonio, TX  University of Iowa Hospitals and Clinics, Iowa City, IA  UC San Diego, San Diego, CA  UCLA, Los Angeles, CA  Methodist Hospital, Houston, TX  Roseman University , UT  Virginia Commonwealth University, VA  University of Rochester Medical Center, Rochester, NY  Virginia Commonwealth University, Richmond, VA