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Potential ASO-based Personalized Treatment for Charcot-Marie-Tooth Disease Type 2S

Author:Tyner, Christina   Smieszek, Sandra   Przychodzen, Bart   Johnson, Caroline   Polymeropoulos, Christos   Birznieks, Gunther   Polymeropoulos, Mihael   

Session Name:S5: ALS and CMT: New Therapeutic Approaches  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S5.009  

Author Institution:Vanda Pharmaceuticals Inc., Washington, DC  Vanda Pharmaceuticals Inc., Washington, DC, DC  

Translating the Effects of CMT2S Variants with Human-on-a-chip Neuromuscular Junction Systems

Author:Johnson, Caroline   Smieszek, Sandra   Przychodzen, Bart   Tyner, Christina   Polymeropoulos, Christos   Birznieks, Gunther   Hagan, Walker   Lenkiu, Leticia   Niccum, Caitlyn   Cannon-Patron, Heather   Brighton, Rocky   Guo, Xiufang   Hawkins, Kenneth   Akanda, Nesar   Hickman, James   Polymeropoulos, Mihael   

Session Name:P2: Neuromuscular and Clinical Neurophysiology (EMG): Spinal Muscular Atrophy and Hereditary Neuropathies  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:P2.014  

Author Institution:Vanda Pharmaceuticals Inc., Washington, DC  Hesperos, Inc., Orlando, FL  NanoScience Technology Center, University of Central Florida, Orlando, FL