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WMS 2026

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05:30 AM
Duration 60mins Hiroshima, Japan
Final results from the JEWELFISH study: 5-year risdiplam treatment in non-treatment-naïve individuals with SMA
Claudia Chiriboga, Claudio Bruno, Dirk Fischer, Eleni Gaki, Eugenio Mercuri, Francesco Muntoni, Francis Warren, Heidemarie Kletzl, Janbernd Kirschner, Ksenija Gorni, Mariacristina Scoto, Pooneh Pordeli, Tina Duong, Valerie Schlegel
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Duration 60mins Hiroshima, Japan
Functional and Safety Outcomes in Older Ambulatory Patients With Duchenne Muscular Dystrophy Treated With Delandistrogene Moxeparvovec
Craig M. McDonald, Erika L. Finanger, Andrés Nascimento, Partha Ghosh, Eugenio M. Mercuri, Vannary Pizzo, Yi Sun, Mark Vivien, Alexander P. Murphy, Marianne Gerber, Pooneh Pordeli, Matthew Furgerson, Crystal Proud
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08:15 AM
Duration 60mins Hiroshima, Japan
Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy: Functional and Safety Outcomes up to 3 Years Post-Infusion in the EMBARK Study
Hirofumi Komaki, Jerry R. Mendell, Francesco Muntoni, Craig M. McDonald, Eugenio M. Mercuri, Emma Ciafaloni, Carmen Leon-Astudillo, Andrés Nascimento, Crystal Proud, Ulrike Schara-Schmidt, Aravindhan Veerapandiyan, Craig M. Zaidman, Matthew Furgerson, Jim Jin, Mark Vivien, Damon R. Asher, Alexander P. Murphy, Carol Reid,* Marianne Gerber, Carmen O. Torre, Marianna Manfrini, Louise R. Rodino-Klapac
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Duration 60mins Hiroshima, Japan
Delandistrogene Moxeparvovec Micro-Dystrophin Expression and Safety in 2–3-year-olds with Duchenne Muscular Dystrophy in ENDEAVOR and ENVOL Studies
Francesco Muntoni, Craig M. McDonald, Andrea Gangfuss, Crystal Proud, Craig M. Zaidman, Laurent Servais, Anne M. Connolly, Patsy Thrasher, Mark Vivien, Alex Haile, Jennifer Thomas-Ahner, Jianfeng Meng, Alexander P. Murphy, Becca B. Zhang, Ewa Karasek, Pooneh Pordeli, Louise R. Rodino-Klapac
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Duration 60mins Hiroshima, Japan
Pooled Safety Analysis from Phase 1 to Phase 3 Clinical Trials of Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy
Crystal Proud, Craig M. McDonald, Eugenio M. Mercuri, Francesco Muntoni, Craig M. Zaidman, Vannary Pizzo, Matthew Furgerson, Jim Jin, Alexander P. Murphy, Mark Vivien, Marianne Gerber, Jerry R. Mendell
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