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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  

Seizures in Childhood Cerebral X-linked Adrenal Leukodystrophy (X-ALD)

Author:Page, Nicole   Nagy, Amanda   Eichler, Florian   Ream, Margie   

Session Name:P2: Child Neurology and Developmental Neurology: Pediatric White Matter  

Topic:Child Neurology and Developmental Neurology  

Program Number:P2.002  

Author Institution:Massachusetts General Hospital, Boston, MA  Nationwide Children'S Hospital/The Ohio State University, Columbus, OH  

Longitudinal Upper Extremity Function in FSHD Monitored by Portable 3D Motion Capture Analyses

Author:Nascimento Andrade, Eduardo Inacio   Ridout, Ben   Penka, Jeff   Stauffer, Jeff   Lowes, Linda   Hayward, Lawrence   

Session Name:P3: Neuromuscular and Clinical Neurophysiology (EMG): Genetic Muscle Diseases  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:P3.006  

Author Institution:Umass Chan Medical School, Worcester, MA  LittleSeed, Inc., Columbus, OH  Nationwide Childrens Hospital, Columbus, OH  University Of Massachusetts Chan Medical School , Worcester, MA