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EPNS 2025

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06:00 AM
Duration 60mins Munich, Germany
Impact of satralizumab▼ on bone strength and muscle function in Duchenne muscular dystrophy (DMD): design of the SHIELD-DMD study
Mercuri E, De Ford C, Guridi M, Estevez-Fraga C, Peck R, Lennon-Chrimes S, Blondeau K, Craggs C, Machado V, Veerapandiyan A, Kostera-Pruszczyk A, Nascimento A, Ward LM, Proud CM

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Duration 60mins Munich, Germany
SUNFISH PARTS 1 AND 2: 5-YEAR EFFICACY AND SAFETY DATA OF RISDIPLAM▼ IN TYPES 2 AND 3 SPINAL MUSCULAR ATROPHY
A Nascimento, J W Day, N Deconinck, E S Mazzone, M Oskoui, K Saito, C Vuillerot, G Baranello, O Boespflug-Tanguy, N Goemans, J Kirschner, A Kostera-Pruszczyk, LServais, K Sully, M Kuthiala, K Gorni, C Martin, W Y Yeung, Renata S Scalco, E Mercuri

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Duration 60mins Munich, Germany
Retrospective assessment of feeding and nutrition after 2 years of risdiplam▼ treatment in younger children with SMA using the CEDAS
G Baranello, E Conway, Y Li, K Gorni

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Duration 60mins Munich, Germany
Clinical Outcomes Among Risdiplam▼-Treated Patients With Spinal Muscular Atrophy (SMA) in the Cure SMA Clinical Data Registry
S Shapouri, T M To, S Whitmire, L Belter, M Schroth, R Schuldt, T Dickendesher

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Duration 60mins Munich, Germany
International evidence-based consensus on the management of gastrointestinal adverse events related to the use of risdiplam▼ in patients with spinal muscular atrophy: methodology
C Hewamadduma and MK Korb on behalf of the SMA Delphi Panel

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Duration 60mins Munich, Germany
3-Year Functional Outcomes of Patients With Duchenne Muscular Dystrophy: Pooled Delandistrogene Moxeparvovec Clinical Trial Data vs External Controls
Alexander P. Murphy; Jerry R. Mendell; Anne M. Connolly; John Day; Craig M. McDonald; Crystal M. Proud; Perry Shieh; Craig Zaidman; Matthew Furgerson; Kai Ding; Carol Reid; Jacob Elkins; Louise Rodino-Klapac

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01:35 PM
Duration 7mins ICM – International Congress Center Munich
Long-Term Functional Outcomes, Safety, and Micro-Dystrophin Expression Following Delandistrogene Moxeparvovec Treatment in DMD: EMBARK 2-Year Results
Francesco Muntoni, Jerry R. Mendell, Craig M. McDonald, Eugenio M. Mercuri, Emma Ciafaloni, Hirofumi Komaki, Carmen Leon-Astudillo, Andrés Nascimento, Crystal Proud, Ulrike Schara-Schmidt, Aravindhan Veerapandiyan, Craig M. Zaidman, Matthew Furgerson, Kai Ding, Preeti Singh, Rachael Potter, Damon R. Asher, Alexander P. Murphy, Carol Reid, Gregory Hooper, Carmen O. Torre, Marianna Manfrini, Jacob S. Elkins, Louise R. Rodino-Klapac

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04:15 PM
Duration 56mins Munich, Germany
Gene therapy in DMD: Reflecting on the now and looking into the future
Dr Astrid Blaschek, Prof. Francesco Muntoni, Dr Felipe Franco da Graça

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11:00 AM
Duration 35mins Munich, Germany
Against the clock: Early interventions in patients with SMA
Prof. Laurent Servais, Prof, Juliana Gurgel Giannetti, Prof. Katharina Vill

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