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Pre-clinical Antisense Oligonucleotide Treatment of CMT2E in a Human Induced Pluripotent Stem Cell (iPSC)-derived Motor Neuron Model

Author:Medina, Jessica   Rebelo, Adriana   Jacobs, Elizabeth   Danzi, Matt   Zuchner, Stephan   Saporta, Mario   

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

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S5.008  

Author Institution:University of Miami, MIAMI, FL  University of Miami School of Medicine, Miami, FL  

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  

Discovering and Rescuing Toxic Autophagy Derangements in CMT4B3 Using a Novel iPSC-derived Peripheral Nervous System Model of MTMR5 Biology

Author:Chua, Jason   Stumpf, Sarah   Dawson, Valina   Dawson, Ted   

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

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S5.010  

Author Institution:Johns Hopkins University, Baltimore, MD  Institute for Cell Engineering, Johns Hopkins University School of Medicine, Baltimore, MD