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WMS 2024

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Coming soon
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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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