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Spinraza in Adults with Spinal Muscular Atrophy (SAS) – 14 month results

Author:Zaidman, Craig   Proud, Crystal   Thonhoff, Jason   Rad, Nassim   Ho, Doreen   Chu, MaryLynn   Ladha, Shafeeq   Crawford, Thomas   Nayar, Shakti   Genge, Angela   Frey, Margaret   Heatwole, Chad   Lew, Daphne   

Session Name:S33: ALS and Motor Neuron Diseases  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S33.010  

Author Institution:Washington University in St Louis, St. Louis, MO  CSG Child & Adolescent Neurology, Norfolk, VA  Houston Methodist Hospital, Houston, TX  University of Washington , Seattle, WA  Massachusetts General Hospital, Boston, MA  NYU-Langone Othopedic Hospital, New York, NY  Barrow Neurological Institute, Phoenix, AZ  Johns Hopkins Hospital, Baltimore, MD  Georgetown, Washington, DC  Mcgill University, Montreal, QC, Canada  Memorial Healthcare , Williamston, MI  University of Rochester Medical Center, Rochester, NY  Washington University School of Medicine, Saint Louis, MO  

One-year Data from ENDEAVOR, a Phase 1b Trial of Delandistrogene Moxeparvovec (SRP-9001) in Patients with Duchenne Muscular Dystrophy (DMD)

Author:Zaidman, Craig   Proud, Crystal   McDonald, Craig   Mason, Stefanie   Guridi, Maitea   Wang, Shufang   Reid, Carol   Darton, Eddie   Wandel, Christoph   Lewis, Sarah   Malhotra, Jyoti   Griffin, Danielle   Potter, Rachael   Rodino-Klapac, Louise   Mendell, Jerry   

Session Name:S48: Therapeutics for Muscle Disease  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S48.003  

Author Institution:Department of Neurology, WUSTL, Washington, MO  Children’s Hospital of the King’s Daughters, Norfolk, VA  UC Davis Health, Sacramento, 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  

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