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Factors Associated with Recurrent Clinical Events among Pediatric Generalized Myasthenia Gravis Patients in the United States

Author:Liu, Zhiwen   Pesa, Jacqueline   Jackson, Louis   Lin, Liang-Yuan   Campbell, Alicia   Govindarajan, Raghav   

Session Name:P8: Child Neurology and Developmental Neurology: Neuromuscular Disorders 2  

Topic:Child Neurology and Developmental Neurology  

Program Number:P8.002  

Author Institution:Johnson & Johnson, Titusville, NJ  Johnson & Johnson, Horsham, PA  HSHS St. Elizabeth Medical Group, O'Fallon, IL  

Dystrophin Deficiency Alters Phospholipid and Sphingolipid Homeostasis in Human Cardiac Organoids

Author:Fontanelli, Lorenzo   Ferri, Carolina   Florentin Suman, Adrian   Ceccherini, Elisa   Recchia, Fabio   Signore, Giovanni   Rocchiccioli, Silvia   

Session Name:P8: Child Neurology and Developmental Neurology: Neuromuscular Disorders 2  

Topic:Child Neurology and Developmental Neurology  

Program Number:P8.003  

Author Institution:Scuola Superiore Sant'Anna, Pisa, Italy  University of Pisa, Italy  Consiglio Nazionale delle Ricerche, Italy  

Treatment of SMA in a Preterm Infant with Elevated Anti-AAV9 Antibody Titers: A Case Report

Author:Lee, Azalea   Verma, Sumit   

Session Name:P8: Child Neurology and Developmental Neurology: Neuromuscular Disorders 2  

Topic:Child Neurology and Developmental Neurology  

Program Number:P8.004  

Author Institution:Emory University School of Medicine, Atlanta, GA  Emory University School of Medicine  Children's Healthcare of Atlanta, Atlanta, GA  

RGX-202, An Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Phase I/II Clinical Data

Author:Veerapandiyan, Aravindhan   Harper, Amy   Iannaccone, Susan   Kuntz, Nancy   Tesi Rocha, Ana Carolina   Boulos, Nidal   Wilson, Catherine   Tsao, Elisa   Gilmor, Michelle   Curtiss, Stacey   Patel, Hiren   Fiscella, Michele   Danos, Olivier   Pakola, Stephen   Dastgir, Janannaz   

Session Name:P8: Child Neurology and Developmental Neurology: Neuromuscular Disorders 2  

Topic:Child Neurology and Developmental Neurology  

Program Number:P8.005  

Author Institution:Arkansas Childrens Hospital/UAMS, Little Rock, AR  Children’s Hospital of Richmond at Virginia Commonwealth University, Richmond, VA  Department of Pediatrics, Dallas, TX  Ann & Robert H Lurie Children'S Hospital of Chicago, Chicago, IL  Stanford University, PALO ALTO, CA  REGENXBIO, Rockville, MD  Regenxbio, Durham, NC  Regenxbio Inc, Rockville, MD  Regenxbio, Irvington, NY  

Clinical and Genetic Spectrum of Duchenne Muscular Dystrophy: Insights from Pakistan’s First National Muscular Dystrophy Registry

Author:Aziz, Bisma   Arif, Ahmed   Malik, Marib Ghulam Rasool   Kirmani, Salman   Ansar, Zeeshan   Nasir, Asghar   Hasan, Zahra   Khan, Sara   

Session Name:P8: Child Neurology and Developmental Neurology: Neuromuscular Disorders 2  

Topic:Child Neurology and Developmental Neurology  

Program Number:P8.006  

Author Institution:Aga Khan University Hospital, Pakistan  The University of North Carolina at Charlotte, NC  Aga Khan University, Karachi, Pakistan  Aga Khan University Hospital, Karachi, Pakistan