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              Ataluren Delays Clinically Meaningful Milestones of Decline in 6WMD in Patients with nmDMD from Study 041, a Phase 3, Placebo-controlled Trial

              Author:Wu, Shiwen   Gulati, Sheffali   Komaki, Hirofumi   Escobar-Cedillo, Rosa E   Kostera-Prusczyk, Anna   Shin, Jin-Hong   Haginoya, Kazuhiro   Penematsa, Vinay   Chou, Connie   Williams, Paula   Werner, Christian   Statland, Jeffrey   

              Session Name:S21: Inherited Myopathies and Neuropathies: New Therapeutic Approaches and Observations  

              Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

              Program Number:S21.001  

              Author Institution:Third Medical Center of PLA, Beijing, China, People's Republic of  All India Institute of Medical Sciences, Ansari Nagar, India  National Center of Neurology ad Psychiatry, Tokyo, Japan  Instituto Nacional de Rehabilitacion, Mexico City, Mexico  Medical University of Warsaw, Warsaw, Poland  Pusan National University Yangsan Hospital, Yangsan, Korea, Republic of  Miyagi Children'S Hospital, Sendai, Japan  PTC Therapeutics, Inc, South Plainfield, NJ  PTC Therapeutics, Inc., South Plainfield, NJ  PTC Therapeutics,GmBH, Frankfurt, Germany  University of Kansas Medical Center, Kansas City, KS  

              Effects of EDG-5506, a Fast Myosin Modulator, on Function and Biomarkers of Muscle Damage in Adults with Becker Muscular Dystrophy

              Author:Phan, Han   Collins, Samuel   Russell, Alan   Barthel, Ben   Thaler, Liz   Kilburn, Nicole   Mancini, Maria   MacDougall, James   Donovan, Joanne   

              Session Name:S21: Inherited Myopathies and Neuropathies: New Therapeutic Approaches and Observations  

              Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

              Program Number:S21.002  

              Author Institution:Rare Disease Research, Atlanta, GA  Edgewise Therapeutics, Christchurch, United Kingdom  Edgewise Therapeutics, Boulder, CO  Edgewise Therapeutics, Topsfield, MA  Edgewise Therapeutics, Boston, MA  Edgewise Therapeutics, Haverhill, MA  

              Effect Size Analysis of Cipaglucosidase Alfa Plus Miglustat Versus Alglucosidase Alfa in ERT-experienced Adults with Late-onset Pompe Disease in PROPEL

              Author:Kushlaf, Hani   Bratkovic, Drago   Byrne, Barry   Claeys, Kristl   Diaz-Manera, Jordi   Dimachkie, Mazen   Kishnani, Priya   Roberts, Mark   Toscano, Antonio   Castelli, Jeff   Holdbrook, Frederick   Das, Sheela   Wasfi, Yasmine   Schoser, Benedikt   Mozaffar, Tahseen   

              Session Name:S21: Inherited Myopathies and Neuropathies: New Therapeutic Approaches and Observations  

              Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

              Program Number:S21.003  

              Author Institution:University of Cincinnati, Cincinnati, OH  PARC Research Clinic, Royal Adelaide Hospital, Adelaide, SA, Australia  University of Florida, Gainesville, FL  Department of Neurology, University Hospitals Leuven, and Laboratory for Muscle Diseases and Neuropathies, Department of Neurosciences, KU Leuven, Leuven, Belgium  John Walton Muscular Dystrophy Research Centre, Newcastle University, Newcastle, United Kingdom  Department of Neurology, University of Kansas Medical Center, Kansas City, KS  Duke University Medical Center, Durham, NC  Salford Royal NHS Foundation Trust, Salford, United Kingdom  ERN-NMD Center for Neuromuscular Disorders of Messina, Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy  Amicus Therapeutics, Inc., Princeton, NJ  Friedrich-Baur-Institute at the Department of Neurology, LMU University Hospital, LMU Munich, Munich, Germany  Department of Neurology, University of California, Irvine, CA  

              Long-term Follow-up Deoxynucleoside Therapy for Late-onset Thymidine Kinase 2 Deficiency Patients

              Author:Durmus, Hacer   Gedikbasi, Asuman   Ceylaner, Serdar   Kiyan, Esen   Parman, Fatma Yesim   

              Session Name:S21: Inherited Myopathies and Neuropathies: New Therapeutic Approaches and Observations  

              Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

              Program Number:S21.004  

              Author Institution:Department of Neurology, Istanbul Faculty of Medicine, Istanbul, Turkey  Istanbul Faculty of Medicine, Istanbul, Turkey  Intergen Genetic Center, Istanbul, Turkey  Istanbul University, Istanbul Faculty of Medicine, Department of Pulmonary Medicine, Istanbul, Turkey, Istanbul, Turkey  

              RGX-202, an Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Clinical Data

              Author:Veerapandiyan, Aravindhan   Rao, Vamshi   Dastgir, Jahannaz   Falabella, Paulo   Pakola, Steve   Rastogi, Sarang   Phillips, Dawn   Wilson, Catherine   Boulos, Nidal   Gilmor, Michelle   Yang, Lin   Patel, Hiren   Fiscella, Michele   Danos, Olivier   

              Session Name:S21: Inherited Myopathies and Neuropathies: New Therapeutic Approaches and Observations  

              Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

              Program Number:S21.005  

              Author Institution:Arkansas Childrens Hospital/UAMS, Little Rock, AR  Ann & Robert H Lurie Children's Hospital, Chicago, IL  REGENEXBIO INC, Rockville, MD  REGENXBIO Inc, Rockville, MD  Regenxbio, Rockville, MD