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GHF-201, A Novel Oral Compound for Adult Polyglucosan Body Disease, Demonstrates Promising Safety and Efficacy in a Compassionate Use Program

Author:Kakhlon, Or   Mordechai, Anat   Garti, Nissim   Gilgun-Sherki, Yossi   Weil, Miguel   Lossos, Alexander   

Session Name:S2: Updates on Motor Neuron and Peripheral Nerve Disorders  

Topic:General Neurology  

Program Number:S2.010  

Author Institution:

A Phase 2/3 Adaptive Design Study of GHF-201 as the First Potential Treatment of Adult Polyglucosan Body Disease

Author:Hirano, Michio   Weil, Miguel   Kakhlon, Or   Mordechai, Anat   Garti, Nissim   Gilgun-Sherki, Yossi   Lossos, Alexander   

Session Name:P5: Neuromuscular and Clinical Neurophysiology (EMG): Motor Neuron Disorders  

Topic:General Neurology  

Program Number:P5.014  

Author Institution: