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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  

Treatments and Outcomes for Patients with Spinal Muscular Atrophy Type 2: Findings from RESTORE Registry

Author:Servais, Laurent   Benguerba, Kamal   De Vivo, Darryl C.   Kirschner, Jan   Muntoni, Francesco   Proud, Crystal   Tizzano, Eduardo   Quijano-Roy, Susana   Desguerre, Isabelle   Saito, Kyoko   Raju, Dheeraj   LaMarca, Nicole   Sun, Rui   Anderson, Fred   Faulkner, Eric   Finkel, Richard   

Session Name:P7: Child Neurology and Developmental Neurology: Spinomuscular Atrophy/Muscular Dystrophies  

Topic:Child Neurology and Developmental Neurology  

Program Number:P7.010  

Author Institution:University of Oxford, Oxford, United Kingdom  Novartis Gene Therapies Switzerland GmbH, Rotkreuz, Switzerland  Columbia University Irving Medical Center, New York, NY  Clinic for Neuropediatrics and Muscle Disease, University Medical Center Freiburg, Freiburg, Germany  The Dubowitz Neuromuscular Centre, University College London, Great Ormond Street Institute of Child Health & Great Ormond Street Hospital and National Institute of Health Research, Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom  Children’s Hospital of the King’s Daughters, Norfolk, VA  Hospital Valle Hebron, Barcelona, Spain  Garches Neuromuscular Reference Center (GNMH), APHP Raymond Poincare University Hospital (UVSQ Paris Saclay), Garches, France  Hôpital Necker Enfants Malades, APHP, Paris, France  Institute of Medical Genetics, Tokyo Women’s Medical University, Tokyo, Japan  Novartis Gene Therapies, Inc., Bannockburn, IL  Center for Outcomes Research, University of Massachusetts Medical School, Worcester, MA  St. Jude Children's Research Hospital, Memphis, TN