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Genotype-phenotype Correlations and Sex Differences in ZC4H2-associated Rare Disorder (ZARD): Results from the ZARD Natural History Study

Author:Peters, Sydney   Sportiello, Kristen   Mandalapu, Shreya   Nguyen, Ashlie   Carrier, Ryan   Dickinson, Carolyn   Paciorkowski, Alexander   Bearden, David   

Session Name:S37: Emerging Therapies in Child Neurology  

Topic:Child Neurology and Developmental Neurology  

Program Number:S37.004  

Author Institution:University of Rochester School of Medicine and Dentistry, Rochester, NY  University of Rochester School of Arts and Sciences, Rochester, NY  Roberts Wesleyan University, Rochester, NY  University of Rochester, Rochester, NY  

Midazolam Versus Ketamine as the First-line Continuous Infusion for Status Epilepticus in Children with Congenital Heart Disease

Author:Javadian, Sam   Keenan, Julia   Liu, Yu-Ting   Voleti, Shruthi   Conley, Carolyn   Staso, Katelyn   Horvat, David   Schlatterer, Sarah   Sansevere, Arnold   Harrar, Dana   

Session Name:S37: Emerging Therapies in Child Neurology  

Topic:Child Neurology and Developmental Neurology  

Program Number:S37.005  

Author Institution:The George Washington University, Washington, DC  Children's National Hospital, Washington, DC  Children'S National Medical Center, Washington, DC  

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  

Mechanisms of Neurodegeneration and Mini-gene Therapeutic Approach for Charcot-Marie-Tooth Disease Type 4B3

Author:Jacobs, Elizabeth   Danzi, Matt   Rebelo, Adriana   Zuchner, Stephan   Saporta, Mario   

Session Name:S37: Emerging Therapies in Child Neurology  

Topic:Child Neurology and Developmental Neurology  

Program Number:S37.007  

Author Institution:University of Miami Miller School of Medicine, Miami, FL  

Assessing Experiences with Trofinetide for Rett Syndrome: Interviews with Caregivers of Patients in Open-label Studies

Author:Bishop, Kathie   Barrett, Amy   Olayinka-Amao, Oyebimpe   Martin, Susan   Doshi, Dilesh   Youakim, James   

Session Name:S37: Emerging Therapies in Child Neurology  

Topic:Child Neurology and Developmental Neurology  

Program Number:S37.008  

Author Institution:Acadia Pharmaceuticals, Inc., San Diego, CA  RTI Health Solutions, Research Triangle Park, NC  Acadia Pharmaceuticals, Inc., Princeton, NJ