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Home Neuroscience MDA 2025
Roche and Genentech at

MDA 2025

March 16 - March 19
Dallas, TX
mdaconference.org
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Agenda
Medical Materials
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
Neuroscience | Duchenne Muscular Dystrophy

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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
Neuroscience | Duchenne Muscular Dystrophy

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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
Neuroscience | Duchenne Muscular Dystrophy

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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
Neuroscience | Spinal Muscular Atrophy

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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
Neuroscience | Spinal Muscular Atrophy

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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
Neuroscience | Duchenne Muscular Dystrophy

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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
Neuroscience | Duchenne Muscular Dystrophy

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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
Neuroscience | Duchenne Muscular Dystrophy

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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
Neuroscience | Duchenne Muscular Dystrophy

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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
Neuroscience | Duchenne Muscular Dystrophy

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Duration 121mins Dallas, TX
Longitudinal Stride-Level Evaluation of Ambulatory Function with Ankle Wearable Technology in Ambulant DMD Patients Below 4 Years Old
Laurent Servais, Margaux Poleur, Guillaume Parinello, Eva Vrščaj, Camille Bisson, Céline Cluzeau, Aurore Daron, Lena Szabo, Damjan Osredkar, Paul Strijbos, Damien Eggenspieler
Neuroscience | Duchenne Muscular Dystrophy

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02:15 PM
Duration 15mins Hilton Anatole
Long-Term Functional Outcomes and Safety of Delandistrogene Moxeparvovec in DMD: EMBARK 2-Year and Pooled 3-Year Analyses
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
Neuroscience | Duchenne Muscular Dystrophy

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02:30 PM
Duration 15mins Hilton Anatole
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
Neuroscience | Duchenne Muscular Dystrophy

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05:00 PM
Duration 15mins Hilton Anatole
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
Neuroscience | Spinal Muscular Atrophy

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Mar 16 / Roche and Genentech
Long-Term Safety and Tolerability of Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy: Phase 1 to Phase 3 Clinical Trials
We report pooled safety outcomes from clinical trials of delandistrogene moxeparvovec in Duchenne muscular dystrophy (DMD) with up to 5 years of follow-up. Among 156 patients, common treatment-related treatment-emergent adverse events (≥15%) included vomiting, nausea, decreased appetite and increased glutamate dehydrogenase, and upper abdominal pain, mostly resolving spontaneously. Treatment-related serious adverse events included liver abnormalities, rhabdomyolysis, vomiting, myocarditis, immune-mediated myositis, nausea, and pyrexia. Safety was consistent across ambulatory and non-ambulatory patients, with no deaths, study discontinuations, or complement activation-related adverse events. These findings support the manageable safety and tolerability profile of delandistrogene moxeparvovec across a broad population of patients with DMD.
Neuroscience Duchenne Muscular Dystrophy MDA-2025

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Mar 16 / Roche and Genentech
Long-term functional outcomes, safety, and micro-dystrophin expression following delandistrogene moxeparvovec treatment in DMD: EMBARK 2-year results
We report 2-year functional and safety outcomes and Week 64 biological outcomes from patients treated with delandistrogene moxeparvovec in Part 1 of EMBARK. Results indicate stabilization or slowing of disease progression compared with well-matched external controls in functional outcomes prognostic for delaying loss of ambulation, with nominally statistically significant differences between the treatment group and external control cohort. Sustained micro‑dystrophin expression and localization to the sarcolemma was observed up to Week 64, and safety was consistent with prior experience.
Neuroscience Duchenne Muscular Dystrophy MDA-2025

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Mar 16 / Roche and Genentech
3-Year Functional Outcomes of Patients With Duchenne Muscular Dystrophy: Pooled Delandistrogene Moxeparvovec Clinical Trial Data vs External Controls
We report 3-year functional outcomes of ambulatory patients (aged 4.0–8.9 years) with Duchenne muscular dystrophy treated with delandistrogene moxeparvovec, compared to propensity-score-matched external controls (EC). Data from 50 treated patients across multiple studies were analyzed using a propensity-score-weighted median regression model and a mixed-effects model for repeated measures. Results showed clinically meaningful stabilization of disease over 3 years in the treatment group versus the ECs, with smaller decline in the North Star Ambulatory Assessment scores and improved performance in timed function tests. These findings support the long-term stabilization or slowing of disease progression with delandistrogene moxeparvovec compared to ECs.
Neuroscience Duchenne Muscular Dystrophy MDA-2025

This website is intended for healthcare professionals outside the United Kingdom (UK) and Australia. Registration status and prescribing information of medicinal products may differ between countries. Please refer to local product information for any medicinal products mentioned on this website.