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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  

Peripheral Neuropathy in AL Amyloidosis: Clinical Presentations and Outcomes of Current Therapies

Author:Chompoopong, Pitcha   Muchtar, Eli   Mauermann, Michelle   

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

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S21.008  

Author Institution:University of Minnesota, Minneapolis, MN  Mayo Clinic, Rochester, MN  

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  

Improved Neuromuscular Junction in R98C Mpz Heterozygous Mice Treated with IFB-088

Author:Bai, Yunhong   Wang, David   Wu, Xingyao   Lamarche, mason   Shy, Michael   

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

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S21.010  

Author Institution:University of Iowa, Iowa City, IA  University Hospitals - Cleveland Medical Center, Cleveland, OH