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Long-term Follow-up Deoxynucleoside Therapy for Late-onset Thymidine Kinase 2 Deficiency Patients

Author:Durmus, Hacer   Gedikbasi, Asuman   Ceylaner, Serdar   Kiyan, Esen   Parman, Fatma Yesim   

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

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S21.004  

Author Institution:Department of Neurology, Istanbul Faculty of Medicine, Istanbul, Turkey  Istanbul Faculty of Medicine, Istanbul, Turkey  Intergen Genetic Center, Istanbul, Turkey  Istanbul University, Istanbul Faculty of Medicine, Department of Pulmonary Medicine, Istanbul, Turkey, Istanbul, Turkey  

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