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Integrated Analyses of Data from Clinical Trials of Delandistrogene Moxeparvovec (SRP-9001) in Duchenne Muscular Dystrophy (DMD)

Author:Proud, Crystal   Zaidman, Craig   Shieh, Perry   McDonald, Craig   Day, John   Mason, Stefanie   Guridi, Maitea   Han, Lixin   Yu, Lixi   Reid, Carol   Darton, Eddie   Wandel, Christoph   Richardson, James   Malhotra, Jyoti   Singh, Tejdip   Rodino-Klapac, Louise   Mendell, Jerry   

Session Name:S48: Therapeutics for Muscle Disease  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S48.006  

Author Institution:Children’s Hospital of the King’s Daughters, Norfolk, VA  Department of Neurology, WUSTL, Washington, MO  UCLA Medical Center, Los Angeles, CA  UC Davis Health, Sacramento, CA  Department of Neurology, Stanford University, Palo Alto, CA  Sarepta Therapeutics, Inc. , Cambridge, MA  F. Hoffmann-La Roche Ltd, Basel, Switzerland  Roche Products Ltd, Welwyn Garden City, United Kingdom  Center for Gene Therapy, Nationwide Children’s Hospital, Columbus, OH  

Long-term Follow-up of Cipaglucosidase Alfa/Miglustat in Ambulatory Patients with Pompe Disease: An Open-label Phase I/II Study (ATB200-02)

Author:Mozaffar, Tahseen   Schoser, Benedikt   Kishnani, Priya   Bratkovic, Drago   Clemens, Paula R.   Goker-Alpan, Ozlem   Ming, Xue   Roberts, Mark   Sivakumar, Kumaraswamy   Van Der Ploeg, Ans   Goldman, Mitchell   Wright, Jacquelyn   Holdbrook, Fred   Jain, Vipul   Sitaraman, Sheela   Wasfi, Yasmine   Byrne, Barry   

Session Name:S48: Therapeutics for Muscle Disease  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S48.007  

Author Institution:University of California, Irvine, Irvine, CA  Friedrich-Baur-Institut, Neurologische Klinik, Ludwig-Maximilians-Universität München, Munich, Germany  Duke University Medical Center, Durham, NC  PARC Research Clinic, Royal Adelaide Hospital, Adelaide, SA, Australia  Department of Neurology, University of Pittsburgh School of Medicine, Division Chief, Neurology, Medical Service Line, VA Pittsburgh Healthcare System, Pittsburgh, PA  Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, VA  Department of Neurology, Rutgers New Jersey Medical School, Newark, NJ  Salford Royal NHS Foundation Trust, Salford, United Kingdom  Neuromuscular Clinic and Research Center, Phoenix, AZ  Erasmus MC University Medical Center, Rotterdam, Netherlands  Amicus Therapeutics, Inc., Philadelphia, PA  University of Florida, Gainesville, FL  

Results from 96 Weeks Open-Label Extension of a Phase 2 Trial of Losmapimod in Subjects with FSHD: ReDUX4

Author:Wang, Leo   Han, Jay   Shoskes, Jennifer   Jiang, John   Tawil, Alrabi   

Session Name:S48: Therapeutics for Muscle Disease  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S48.008  

Author Institution:University of Washington, Seattle, WA  UCI Medical Center, Orange, CA  Fulcrum Therapeutics, Cambridge, MA  University of Rochester Medical Center, Rochester, NY  

Results from a 36-Week Open-Label Study of Recombinant Human Growth Hormone and Testosterone in Facioscapulohumeral Muscular Dystrophy (FSHD)

Author:Heatwole, Chad   Luebbe, Elizabeth   Hamel, Johanna   Mongiovi, Philip   Ciafaloni, Emma   Dilek, Nuran   Martens, William   Weber, David   Hani, Rashid   Allen, Jamie   Smith, Claire   Howell, Samantha   Rosero, Spencer   Eichinger, Katy   Baker, Linday   Dekdebrun, Jeanne   Hilbert, Jim   Varma, Anika   McDermott, Michael   Thornton, Charles   Moxley, Richard   

Session Name:S48: Therapeutics for Muscle Disease  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S48.009  

Author Institution:University of Rochester Medical Center, Rochester, NY  Children's Hospital of Philadelphia, Philadelphia, PA  University of Rochester Medical Center, Baltimore, MD  

Annualized Rates of Change from A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week Study of Losmapimod in Subjects with FSHD: ReDUX4

Author:Tawil, Alrabi   

Session Name:S48: Therapeutics for Muscle Disease  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S48.010  

Author Institution:University of Rochester Medical Center, Rochester, NY