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Roche and Genentech at

MDA 2025

We will be contributing to topics related to
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11:00 PM
Duration 120mins Dallas, TX
Assessing biomarkers of bone metabolism and the role of the IL-6 signalling pathway in patients with Duchenne Muscular Dystrophy
M Guridi, C De Ford, C Fruechtenicht, C Gee See, R Houghton, Y Chen, AP Murphy, C Wood, L Ward, N Crabtree, EM Mercuri, H McMillan
Duration 120mins Dallas, TX
Natural history of bone health in Duchenne Muscular Dystrophy: A systematic review and implications for the design of a clinical trial
C De Ford, M Guridi, Y Chen, A P. Murphy, C Wood, H McMillan, E M. Mercuri, N Crabtree, L Ward
Duration 120mins Dallas, TX
Impact of satralizumab▼ on bone strength and muscle function in Duchenne muscular dystrophy (DMD): design of the SHIELD-DMD study
Proud CM, 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, Mercuri E
Duration 120mins Dallas, TX
Real-world risdiplam▼ effectiveness in adults with spinal muscular atrophy (SMA) from the Pediatric Neuromuscular Clinical Research (PNCR) registry
D Jayaraman, S Roumpanis, W Martens, T Dickendesher, S Shapouri, B Darras, RS Finkel, M Hirano, Z Zolkipli-Cunningham, A Gershon, on behalf of the PNCR network
Duration 120mins Dallas, TX
SUNFISH Parts 1 and 2: 5-year efficacy and safety data of risdiplam▼ in Types 2 and 3 spinal muscular atrophy (SMA)
L Servais, JW Day, N Deconinck, E Mazzone, A Nascimento, M Oskoui, K Saito, C Vuillerot, G Baranello, O Boespflug-Tanguy, N Goemans, J Kirschner, A Kostera-Pruszczyk, K Sully, M Kuthiala, K Gorni, C Martin, WY Yeung, R Scalco, E Mercuri, on behalf of the SUNFISH Study Group
Duration 121mins Dallas, TX
Muscle MRI outcomes in patients with Duchenne Muscular Dystrophy treated with delandistrogene moxeparvovec: Findings from EMBARK Part 1
K Vandenborne, G Walter, V Straub, R Willcocks, S Forbes, S Ennamuri, K Ding, C Reid, A Murphy, M Manfrini, J Elkins, L Rodino-Klapac4
Duration 121mins Dallas, TX
Long-term functional outcomes, safety, and micro-dystrophin expression following delandistrogene moxeparvovec treatment in DMD: EMBARK 2-year results
J R. Mendell, F Muntoni, C M. McDonald, E M. Mercuri, E Ciafaloni, H Komaki, C Leon-Astudillo, A Nascimento, C Proud, U Schara-Schmidt, A Veerapandiyan, C M. Zaidman, M Furgerson, K Ding, P Singh, R Potter, D R. Asher, A P. Murphy, C Reid, G Hooper, C O. Torre, M Manfrini, J S. Elkins, L R. Rodino-Klapac, on behalf of the EMBARK Study Group
Duration 121mins Dallas, TX
3-Year Functional Outcomes of Patients With Duchenne Muscular Dystrophy: Pooled Delandistrogene Moxeparvovec Clinical Trial Data vs External Controls
Jerry Mendell, Anne M. Connolly, John Day, Craig McDonald, Crystal Proud, Perry Shieh, Craig Zaidman, Matthew Furgerson, Kai Ding, Carol Reid, Alexander P. Murphy, Jacob S. Elkins, Louise Rodino-Klapac
Duration 121mins Dallas, TX
Assessment of Cardiac Outcomes in Delandistrogene Moxeparvovec Clinical Trials for Duchenne Muscular Dystrophy
Aravindhan Veerapandiyan, John Bourke, John Day, Craig McDonald, Jerry Mendell, Jonathan Soslow, Craig Zaidman, Stefanie Mason, Jianfeng Meng, Mark Vivien, Tao Niu, Alexander P. Murphy, Christoph Wandel, James Richardson
Duration 121mins Dallas, TX
Long-Term Safety and Tolerability of Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy: Phase 1 to Phase 3 Clinical Trials
Jerry Mendell, Craig McDonald, Eugenio Mercuri, Francesco Muntoni, Craig Zaidman, Sachi Dharia, Stefanie Mason, Jianfeng Meng, Alexander P. Murphy, Emanuel Palatinsky, Carol Reid, Christoph Wandel, Crystal Proud
02:15 PM
Duration 15mins Dallas, TX
Long-term functional outcomes and safety of delandistrogene moxeparovec in DMD: EMBARK 2-year and Pooled 3-year analyses's
C Proud, J R. Mendell, F Muntoni, C M. McDonald, E M. Mercuri, E Ciafaloni, H Komaki, C Leon-Astudillo, A Nascimento, C Proud, U Schara-Schmidt, A Veerapandiyan, C M. Zaidman, M Furgerson, K Ding, P Singh, R Potter, D R. Asher, A P. Murphy, C Reid, G Hooper, C O. Torre, M Manfrini, J S. Elkins, L R. Rodino-Klapac, on behalf of the EMBARK Study Group
02:30 PM
Duration 15mins Dallas, TX
Muscle MRI outcomes in patients with Duchenne Muscular Dystrophy treated with delandistrogene moxeparvovec: Findings from EMBARK Part 1
K Vandenborne, G Walter, V Straub, R Willcocks, S Forbes, S Ennamuri, K Ding, C Reid, A Murphy, M Manfrini, J Elkins, L Rodino-Klapac4
05:00 PM
Duration 15mins Dallas, TX
RAINBOWFISH: 2-year efficacy and safety data of risdiplam▼ in infants with presymptomatic spinal muscular atrophy (SMA)
RS Finkel, MA Farrar, L Servais, D Vlodavets, E Zanoteli, M Al-Muhaizea, APQC Araujo, L Nelson, B Jaber, K Gorni, H Kletzl, L Palfreeman, E Gaki, M Rabbia, D Summers, P Fontoura, E Bertini
Coming soon