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RGX-202, an Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Clinical Data

Author:Veerapandiyan, Aravindhan   Rao, Vamshi   Dastgir, Jahannaz   Falabella, Paulo   Pakola, Steve   Rastogi, Sarang   Phillips, Dawn   Wilson, Catherine   Boulos, Nidal   Gilmor, Michelle   Yang, Lin   Patel, Hiren   Fiscella, Michele   Danos, Olivier   

Session Name:S21: Inherited Myopathies and Neuropathies: New Therapeutic Approaches and Observations  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S21.005  

Author Institution:Arkansas Childrens Hospital/UAMS, Little Rock, AR  Ann & Robert H Lurie Children's Hospital, Chicago, IL  REGENEXBIO INC, Rockville, MD  REGENXBIO Inc, Rockville, MD  Regenxbio, Rockville, MD  

REMEDY: A Novel CRISPR-based Allele Specific Approach Corrects VCP Mutations

Author:D'Ambrosio, Eleonora   Ozes-Ak, Burcak   Sahenk, Zarife   Mendell, Jerry   Naeimi Kararoudi, Meisam   

Session Name:S21: Inherited Myopathies and Neuropathies: New Therapeutic Approaches and Observations  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S21.006  

Author Institution:Nationwide Children's Hospital, Columbus, OH  

Peripheral Nervous System Involvement of Hereditary Transthyretin Amyloidosis in the United States: A Multi-center Perspective

Author:Ilieva, Hristelina   Eyer, James   Desai, Urvi   Peltier, Amanda   

Session Name:S21: Inherited Myopathies and Neuropathies: New Therapeutic Approaches and Observations  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S21.007  

Author Institution:ALS Weinberg Clinic, Philadelphia, PA  Meharry Medical School , Nashville, TN  Dept of Neurology, CMC, Charlotte, NC  Vanderbilt University, Nashville, TN  

PAK2 Is Necessary for Myelination in the Peripheral Nerve System

Author:Hu, Bo   Moiseev, Daniel   Schena, Isabella   Faezov, Bulat   Dunbrack, Roland   Chernoff, Jonathan   Li, Jun   

Session Name:S21: Inherited Myopathies and Neuropathies: New Therapeutic Approaches and Observations  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S21.009  

Author Institution:Houston Methodist Hospital, Houston, TX  Wayne State University School of Medicine, Detroit, MI  Fox Chase Cancer Center, Philadelphia, PA  

Randomized Controlled Trial of Lidocaine Occipital Nerve Blocks to Treat Status Migrainosus in Children/Adolescents

Author:Szperka, Christina   Marquez de Prado, Blanca   Hsu, Jesse   Gelfand, Amy   Haagen, Dana   Kellier, Danielle   Dlugos, Dennis   Raj, Nichelle   Hershey, Andrew   Farrar, John   

Session Name:S22: Advances in Migraine and Therapeutics  

Topic:Headache  

Program Number:S22.001  

Author Institution:Children's Hospital of Philadelphia & Perelman School of Medicine at the Univ of PA, Philadelphia, PA  Children's Hospital of Philadelphia, Philadelphia, PA  University of Pennsylvania, Philadelphia, PA  UCSF, San Francisco, CA  Fox Chase Cancer Center, Philadelphia, PA  Cincinnati Childrens Hospital Medical Center, Cincinnati, OH