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90-month Muscle Function and Biomarker Outcomes With Cipaglucosidase Alfa Plus Miglustat (Cipa+Mig) in Adults With Pompe Disease in ATB200-02, an Open-label Phase I/II Study

Author:di Ronza, Alberto   Bratkovic, Drago   Byrne, Barry J.   Clemens, Paula R.   Goker-Alpan, Ozlem   Kishnani, Priya S.   Mozaffar, Tahseen   Roberts, Mark   Van Der Beek, Nadine AME   Amon, Farah   Fox, Brian   Holdbrook, Fred   Jain, Vipul   Schoser, Benedikt   

Session Name:S7: Updates on Muscle Disorders  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S7.006  

Author Institution:Amicus Therapeutics, Inc., Princeton, NJ, USA, Rosharon, TX  PARC Research Clinic, Royal Adelaide Hospital, Adelaide, SA, Australia, Adelaide, Australia  University of Florida, Gainesville, FL, USA, Gainesville, FL  Department of Neurology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA, Pittsburgh, PA  ysosomal and Rare Disorders Research and Treatment Center, Fairfax, VA, USA, Fairfax, VA  Duke University Medical Center, Durham, NC, USA, Durham, NC  Department of Neurology, University of California, Irvine, CA, USA, Orange, CA  Salford Royal NHS Foundation Trust, Salford, UK, Salford, United Kingdom  Erasmus MC University Medical Center, Rotterdam, Netherlands, Rotterdam, Netherlands  Amicus Therapeutics, Inc., Princeton, NJ, USA, Princeton, NJ  Friedrich-Baur-Institute at the Department of Neurology, LMU University Hospital, LMU Munich, Munich, Germany, Munich, Germany  

Phase II/III Study of Ulviprubart in Inclusion Body Myositis: Study Design, Patient Characteristics, and Topline Results

Author:Goyal, Namita A.   Machado, Pedro M.   Ascherman, Dana P.   Benveniste, Olivier   Beydoun, Said R.   Brannagan, Thomas H.   Christopher-Stine, Lisa   Collins, Michael P.   De Bleecker, Jan L.   Dimachkie, Mazen   Dixon, Stacy   Fernandez, David R.   Freimer, Miriam   Geiger, Christopher   Gonzalez-Perez, Paloma   Goyal, Neelam   Gwathmey, Kelly   Heitzman, Daragh   Henderson, Robert D.   Hodson-Webb, Lisa D.   Hussain, Yessar   Korngut, Lawrence   Liang, Christina   Lilleker, James B.   Lloyd, Thomas   Mohassel, Payam   Naddaf, Elie   Needham, Merrilee   Piccione, Ezequiel   Quinn, Colin C.   Rosow, Laura   Roy, Bhaskar   Ruck, Tobias   Schmidt, Jens   Seth, Arjun   Shah, Jaimin   Shaibani, Aziz   Shieh, Perry B.   Simmons, Zachary   Sivakumar, Kumaraswamy   Wang, Leo H.   Weihl, Conrad C.   Wilkins, H. Jeffrey   Amato, Anthony   

Session Name:S7: Updates on Muscle Disorders  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S7.007  

Author Institution:Department of Neurology, University of California Irvine, Orange, CA, USA, Orange, CA  Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, University College London, London, UK  NIHR University College London Hospitals Biomedical Research Centre, University College London Hospitals NHS Foundation Trust, London, UK, London, United Kingdom  Department of Medicine, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA, Pittsburgh, PA  Department of Internal Medicine and Clinical Immunology, Pitié-Salpêtrière University Hospital, Sorbonne Université, AP-HP, Paris, France, Paris, France  University of Southern California, Los Angeles, CA, USA, Los Angeles, CA  Neurological Institute, Columbia University Medical Center, New York, NY, USA, New York, NY  Johns Hopkins University School of Medicine, Baltimore, MD, USA, Baltimore, MD  Medical College of Wisconsin, Milwaukee, WI, USA, Milwaukee, WI  Department of Neurology and Neuromuscular Reference Center, Ghent University Hospital, Ghent, Belgium, Ghent, Belgium  Department of Neurology, The University of Kansas Medical Center, Kansas City, KS, USA, Kansas City, KS  University of Colorado, Aurora, CO, USA, Aurora, CO  Department of Rheumatology, Hospital for Special Surgery, New York, NY, USA, New York, NY  Department of Neurology, The Ohio State Wexner Medical Center, Columbus, OH, USA, Columbus, OH  University Hospitals-Cleveland Medical Center, Case Western Reserve University, Cleveland, OH, USA, Cleveland, OH  Department of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA, Boston, MA  Stanford Neuroscience Health Center, Palo Alto, CA, USA, Palo Alto, CA  Department of Neurology, Virginia Commonwealth University, Richmond, VA, USA, Henrico, VA  Texas Neurology, Dallas, TX, USA, Dallas, TX  Department of Neurology, Royal Brisbane and Women's Hospital, Brisbane, Australia  Centre for Clinical Research, University of Queensland, Brisbane, Australia, Brisbane, Australia  Neuromuscular Division/Department of Neurology, Duke University, Durham, NC, USA, Durham, NC  Austin Neuromuscular Center, Austin, TX, USA, Austin, TX  Hotchkiss Brain Institute, Department of Clinical Neurosciences, Cumming School of Medicine, University of Calgary, Alberta, Canada, Calgary, AB, Canada  Faculty of Medicine and Health, University of Sydney, Camperdown, New South Wales, Australia  Department of Neurology, Royal North Shore Hospital, Northern Sydney Local Health District, Sydney, New South Wales Australia  Neuroscience Research Australia, Sydney, New South Wales, Australia, Australia  Manchester Centre for Clinical Neurosciences, Northern Care Alliance NHS Foundation Trust, Salford, UK  The University of Manchester, Manchester, UK, United Kingdom  Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, USA  Department of Neurology, Baylor College of Medicine, Houston, TX, USA  Department of Clinical Research, Imricor Medical Systems, Burnsville, MN, USA, Houston, TX  Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, USA, Baltimore, MD  Department of Neurology, Mayo Clinic, Rochester, MN, USA, Rochester, MN  Perron Institute, QEII Medical Centre, Nedlands, Western Australia, Australia  University of Notre Dame, Perth, Western Australia, Australia  Fiona Stanley Hospital, Perth, Western Australia, Australia  Murdoch University, Perth, Western Australia, Australia, Murdoch, Australia  Department of Neurology, University of Nebraska Medical Center, Omaha, NE, USA, Omaha, NE  Neuromuscular Division, Neurology Department, University of Pennsylvania, Philadelphia, PA, USA, PA  University of California San Francisco, San Francisco, CA, USA, San Francisco, CA  Department of Neurology, Yale School of Medicine, New Haven, CT, USA, New Haven, CT  Ruhr University Bochum, BG-University Hospital Bergmannsheil Department of Neurology, Bochum, Germany  Heimer Institute for Muscle Research, BG-University Hospital Bergmannsheil Department of Neurology, Bochum, Germany, Düsseldorf, Germany  Department of Neurology, Neuromuscular Center, University Medical Center Göttingen, Göttingen, Germany  Department of Neurology and Pain Treatment, Neuromuscular Center, Center for Translational Medicine, Immanuel University Hospital, Brandenburg Medical School, Rüdersdorf bei Berlin, Germany, Rüdersdorf bei Berlin, Germany  Department of Neurology, Northwestern Memorial Hospital, Chicago, IL, USA, Chicago, IL  Department of Neurology, Mayo Clinic, Jacksonville, FL, USA, Jacksonville, FL  Nerve and Muscle Center of Texas, Houston, TX, USA, Houston, TX  Department of Pediatrics, University of California Los Angeles, Los Angeles, CA, USA, Los Angeles, CA  Department of Neurology, Penn State University College of Medicine, Hershey, PA, USA, Hershey, PA  The Neuromuscular Research Centre and Neuromuscular Clinic of Arizona, Phoenix, AZ, USA, Paradise Valley, AZ  Department of Neurology, University of Washington, Seattle, WA, USA, WA  Department of Neurology, Washington University in St Louis, St Louis, MO, USA, MO  Abcuro, Inc., Newton, MA, USA, MA  Brigham and Women’s Hospital, Harvard Medical School, Boston, MA, USA, Boston, MA  

Current Abilities Score (CAS) is a Valid and a Reliable PROM in FSHD: Data From MOVE Natural History Study

Author:Hewamadduma, Channa   Walker, Michaela   Street, jonathan   Katz, Natalie   Eichinger, Kate   Di Pace, Filipe   sasidharan, sandhya   lowes, linda   Ammoreno, Cristiane   Statland, Jeffrey   

Session Name:S7: Updates on Muscle Disorders  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S7.008  

Author Institution:Sheffield Institute for Translational Neurosciences (SITRAN), University of Sheffield, Sheffield, United Kingdom  University of Kansas Medical Center, Fairway, KS  Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, United Kingdom  Duke University, Durham, NC  University of Rochester, NY  Universidade de São Paulo , Brazil  University of Kansas Medical Center, KS  Sarepta, MA  Universidade de São Paulo , São Paulo , Brazil  University of Kansas Medical Center, Kansas City, KS  

Integration of Muscle Fat Fraction and Apparent Diffusion Coefficient Analyses to Track Disease Progression in Oculopharyngeal Muscular Dystrophy Using Whole-body Quantitative MRI

Author:Satarkar, Tanay   Smith, Ian   Melkus, Gerd   Sampaio, Marcos   Esttaifo, Fadi   Meier-Ross, Kaitlynn   Khan, Sono   Warman, Jodi   

Session Name:S7: Updates on Muscle Disorders  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S7.009  

Author Institution:University of Ottawa, Ottawa, ON, Canada  Ottawa hospital research institute, Ottawa, ON, Canada  Ottawa hospital research institute, ON, Canada  University of Ottawa, ON, Canada  The Ottawa Hospital, Ottawa, ON, Canada  

Early Fatal Myositis/Myasthenia Induced by Immune Checkpoint Inhibitors: A Nationwide Retrospective Study

Author:Velasco Fargas, Roser   Cabrera-Maqueda, Jose Maria   Fonseca, Elianet   Marco, Carla   Chico-Garcia, Juan Luis   Llaurado, Arnau   Gomez, Francisco Javier   Izquierdo, Cristina   Cabello, Francisco Javier   del Pino, Laura   Cortes-Vicente, Elena   Horga, Alejandro   Gallego, Jaime   Jauregui, Carlota   Caldu, Rocio   Pelayo, Ana Lara   Ros, Marta   Hernandez Tost, Hugo   Alvarez-Troncoso, Jorge   alaña, Monica   Torne, Laura   Gutierrez-Gutierrez, Gerardo   Gonzalez-Mingot, Cristina   Garcia-Yepes, Marta   Chavarria, Alba   Riancho, Javier   Sanchez-Vizcaino, Cristina   Rodrigo, Patricia   Besora, Sarah   Cabezudo-García, Pablo   Moris, German   Guijarro, Monica   Macias, Adrian   Muñiz, Sergio   Carvajal, Alejandra   Baron, Manuel   Pouso, Jessica   Boix, Almudena   Dalmau, Josep   Bruna, Jordi   Martinez-Hernandez, Eugenia   

Session Name:S7: Updates on Muscle Disorders  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S7.010  

Author Institution:Catalan Institute of Oncology, Barcelona, Spain  Hospital Clinic de Barcelona, Spain  Hospital Universitario de Bellvitge, Spain  Hospital Ramon y Cajal , Madrid, Spain  Hospital Vall d'Hebron, Barcelona, Spain  Hospital Virgen del Rocio, Sevilla, Spain  Hospital Germans Tries i Pujol, Badalona, Spain  Hospital La Fe, Valencia, Spain  Hospital Gregorio Marañon, Madrid, Spain  Hospital de la Santa Creu i Sant Pau, Barcelona, Spain  Hospital Clinico de Madrid, Spain  Clinica Universitaria de Navarra, Spain  Hospital Universitario de Donostia, San Sebastian, Spain  Hospital Lozano Blesa, Zaragoza, Spain  Hospital Marques de Valdecilla, Santander, Spain  Hospital Parc Tauli, Sabadell, Spain  Hospital Nuestra Señora de la Candelaria, Santa Cruz de Tenerife, Spain  Hospital La Paz, Madrid, Spain  Complejo Asistencial Universitario de Salamanca, SALAMANCA, Spain  Hospital Universitario de Navarra, Pamplona, Spain  Hospital Universitario Infanta Sofía, Madrid, Spain  Hospital Arnau de Vilanova, Lleida, Spain  Complejo Hospitalario Universitario Insular Materno Infantil, Las Palmas de Gran Canaria, Spain  Hospital Clinico Universitario de Valladolid, Valladolid, Spain  Hospital Sierrallana, Torrelavega, Spain  Hospital General Universitario Santa Lucía, Cartagena, Spain  Hospital Universitario Basurto, Bilbao, Spain  Hospital Mutua de Terrassa, Terrassa, Spain  Hospital Regional Universitario de Malaga, Málaga, Spain  Hospital Central de Asturias, Oviedo, Spain  Hospital Universitario de Lugo, Spain  Hospital del Mar, Barcelona, Spain  Hospital 12 de Octubre, Madrid, Spain  Hospital Universitario Virgen de las Nieves, Granada, Spain  Hospital Universitario Fundacion Alcorcon, Madrid, Spain  Complejo Hospitalario Universitario de Pontevedra, Pontevedra, Spain  Hospital Josep Trueta, Girona, Spain  Institut d'Investigacions Biomediques August Pi i Sunyer - Caixa Research Institute, Barcelona, Spain