WMS 2024

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Agenda

Medical Materials

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

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

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

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

Duration 1hr Prague, Czechia

Investigating the data landscape of Duchenne Muscular Dystrophy: Answering your research questions with the right data source(s)

H Furby, A Simpson, M Johnson, J Signorovitch,YMoride, G Castillon

Neuroscience | Duchenne Muscular Dystrophy

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

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

Duration 1hr Prague, Czechia

Safety and efficacy of delandistrogene moxeparvovec versus placebo in Duchenne muscular dystrophy (EMBARK): Pivotal Phase 3 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

Duration 1hr Prague, Czechia

Expression and safety with delandistrogene moxeparvovec gene therapy for Duchenne Muscular Dystrophy in a broad population: Phase 1b ENDEAVOR trial

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

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

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

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

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

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

05:45 AM

Duration 15mins Congress Hall

Muscle MRI outcomes in delandistrogene moxeparvovec-treated patients with Duchenne muscular dystrophy 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

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

Coming soon

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WMS 2024