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Miglustat: A First-In-Class Enzyme Stabilizer for Late-Onset Pompe Disease

Author:Mozaffar, Tahseen   Byrne, Barry J.   Dimachkie, Mazen   Hopkin, Robert J.   Kishnani, Priya   Roberts, Mark   Schoser, Benedikt   Van Der Ploeg, Ans   Brudvig, Jon   Fox, Brian   Holdbrook, Frederick   Jain, Vipul   Johnson, Franklin   Zhang, Jennifer   Parenti, Giancarlo   

Session Name:P10: General Neurology: New, Potential, and Innovative Treatments 2  

Topic:General Neurology  

Program Number:P10.012  

Author Institution:University of California, Irvine, Irvine, CA  University of Florida, Gainesville, Gainesville, FL  University of Kansas Medical Center, Kansas City, KS  Cincinnati Children's Hospital Medical Center, Cincinnati, OH  Duke University Medical Center, Durham, NC  Salford Royal NHS Foundation Trust, Salford, United Kingdom  LMU University Hospital, LMU Munich, Munich, Germany  Erasmus MC University Medical Center, Rotterdam, Netherlands  Amicus Therapeutics, Inc., Princeton, NJ  University of Naples Federico II, Naples, Italy