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Home Neuroscience WMS 2024
Roche and Genentech at

WMS 2024

October 08 - October 12
Prague, Czechia
wms2024.com
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Medical Materials
Agenda
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12:30 PM
Duration 1hr Prague, Czechia
Retrospective assessment of feeding and nutrition after 2 years of risdiplam▼ treatment in children with Type 1 spinal muscular atrophy (SMA) using a novel scale
G Baranello, E Conway, Y Li, K Gorni
Neuroscience | Spinal Muscular Atrophy

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Duration 1hr Prague, Czechia
Assessing biomarkers of bone metabolism and the role of the IL-6 signalling pathway in patients with Duchenne Muscular Dystrophy
C De Ford, M Guridi, C Fruechtenicht, C Gee See, R Houghton, Y Chen, AP Murphy, C Wood, LM Ward, N Crabtree, EM. Mercuri, H McMillan
Neuroscience | Duchenne Muscular Dystrophy

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Duration 1hr Prague, Czechia
Natural history of bone health in Duchenne Muscular Dystrophy: A systematic review and implications for design of clinical trial
C De Ford, M Guridi, Y Chen, AP Murphy, C Wood, H McMillan, EM Mercuri, N Crabtree, LM Ward
Neuroscience | Duchenne Muscular Dystrophy

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Duration 1hr Prague, Czechia
A qualitative evaluation of meaningful change on the North Star Ambulatory Assessment and Performance of Upper Limb in Duchenne muscular dystrophy
AP Murphy, T Ciobanu, E Davies, A Gillman, L Barrett, A Johnson, J Mills, P Heinrich, K Przydzial, B Ewens, G Vandenberg, S Cano, A Mayhew
Neuroscience | Duchenne Muscular Dystrophy

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Duration 1hr Prague, Czechia
Investigating the data landscape of Duchenne Muscular Dystrophy: Answering your research questions with the right data source
H Furby, A Simpson, M Johnson, J Signorovitch,YMoride, G Castillon
Neuroscience | Duchenne Muscular Dystrophy

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Duration 1hr Prague, Czechia
Longitudinal evaluation of ambulatory function with ankle wearable technology in ambulant DMD
M Poleur, G Parinello, E Vrščaj, M Kumhera, C Bisson, K Aragon-Gawińska, C Anghelescu, A Daron, L Szabo, M Leanca, A Mirea, SKodsy, A Saleh, D Osredkar, J, A Potulska-Chromik, N Butoianu,D Eggenspieler2 P Strijbos and L Servais
Neuroscience | Duchenne Muscular Dystrophy

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03:15 PM
Duration 1hr Prague, Czechia
Cardiac MRI outcomes in patients with Duchenne muscular dystrophy treated with delandistrogene moxeparvovec: findings from EMBARK Part 1
G Walter, K Vandenborne, J Bourke, J Soslow, S Mason, E Palatinsky, C Wandel, W Zhang, C Reid, AP Murphy, M Manfrini, J Richardson, LR. Rodino- Klapac
Neuroscience | Duchenne Muscular Dystrophy

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Duration 1hr Prague, Czechia
Safety and efficacy of delandistrogene moxeparvovec versus placebo in Duchenne Muscular Dystrophy: Phase 3 EMBARK primary results
Mendell JR, Muntoni F, McDonald CM, Mercuri EM, Ciafaloni E, Komaki H, Leo-Astudillo C, Nascimento A, Proud C, Schara-Schmidt U, Veerapandiyan A, Zaidman CM, Guridi M, Murphy AP, Reid C, Wandel C, Asher DR, Darton E, Mason S, Potter RA, Singh T, Zhang W, Fontoura P, Elkins JS, Rodino-Klapac LR
Neuroscience | Duchenne Muscular Dystrophy

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Duration 1hr Prague, Czechia
Micro-dystrophin expression and safety with delandistrogene moxeparvovec gene therapy for DMD in a broad population: Phase 1b trial (ENDEAVOR)
C Proud, CM Zaidman, CM McDonald, JW Day,P Thrasher, DR Asher, AP Murphy, M Guridi, K Ding, C Reid, S Lewis, P Magistrado-Coxen, E Palatinsky, C Wandel, RA Potter, LR Rodino-Klapac, JR Mendell
Neuroscience | Duchenne Muscular Dystrophy

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05:00 PM
Duration 1min Prague, Czechia
Impact of disease-modifying therapies on bulbar function in children and adults living with SMA: Learnings and limitations
Giovanni Baranello, Katlyn McGrattan, Giorgia Coratti
Neuroscience | Spinal Muscular Atrophy

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12:15 PM
Duration 1hr Prague, Czechia
Demographic and clinical characteristics of risdiplam▼-treated and untreated adult patients with spinal muscular atrophy (SMA)
K Gorni, M Batech, CJ Guittari, A Carmona, SY Lee, A Poll, CS Sutherland, C Marini-Bettolo, MC Walter, M Jagut, J Haberlov, V Hodgkinson, E Yiu, LB Murphy, TREAT-NMD Global Data systems Oversight Committee (TGDOC), TREAT-NMD Global Registry Network for SMA, H Gordish-Dressman, A Simpson
Neuroscience | Spinal Muscular Atrophy

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Duration 1hr Prague, Czechia
Burden of illness for male patients with Duchenne muscular dystrophy (DMD) in a real-world setting, a Swedish registry study
H Furby, DM Boudreau, C De Ford, A-B Ekstrom, A-K Kroksmark, N Stelmaszuk, E Streja, J Wojtowicz, Q Yang6
Neuroscience | Duchenne Muscular Dystrophy

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01:45 PM
Duration 1hr Prague, Czechia
Real-world treatment with risdiplam▼ in adults with spinal muscular atrophy (SMA): US multicentre study
K Gorni, CJ Guittari, S Candrilli, LS Miles, A Simpson, S Shapouri
Neuroscience | Spinal Muscular Atrophy

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Duration 1hr Prague, Czechia
Fertility outcomes in risdiplam▼-treated male patients with spinal muscular atrophy: a multicenter case series
M Erdler, S Coskery, MR Frey, MA Lopez
Neuroscience | Spinal Muscular Atrophy

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Duration 1hr Prague, Czechia
Long-term safety and tolerability of delandistrogene moxeparvovec in Duchenne muscular dystrophy: phase 1 to phase 3 clinical trials
C Proud, CM McDonald, EM Mercuri, F Muntoni, CM Zaidman, S Dharia, S Mason, J Meng, AP Murphy, E Palatinsky, C Reid, C Wandel, JR Mendell
Neuroscience | Duchenne Muscular Dystrophy

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05:45 AM
Duration 15mins Congress Hall
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, W Zhang, C Reid, AP Murphy, M Manfrini, JS Elkins, LR Rodino-Klapac
Neuroscience | Duchenne Muscular Dystrophy

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06:30 AM
Duration 15mins Congress Hall
RAINBOWFISH: 2-year efficacy and safety data of risdiplam▼ in infants with presymptomatic SMA
L Servais, RS Finkel, MA Farrar, D Vlodavets, E Zanoteli, M Al-Muhaizea, APQC Araújo, L Nelson, B Jaber, K Gorni, H Kletzl, L Palfreeman, E Gaki, M Rabbia, D Summers, P Fontoura, E Bertini, on behalf of the RAINBOWFISH Study Group
Neuroscience | Spinal Muscular Atrophy

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