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Home Neuroscience WMS 2025
Roche and Genentech at

WMS 2025

October 07 - October 11
Vienna, Austria
wms2025.com
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Medical Materials
Agenda
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12:15 PM
Duration 60mins Vienna, Austria
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 Shieh6; Craig Zaidman; Matthew Furgerson; Kai Ding; Carol Reid8; Alexander P. Murphy; Louise Rodino-Klapac
Neuroscience | Duchenne Muscular Dystrophy

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12:30 PM
Duration 60mins Vienna, Austria
HINALEA 2: Baseline observations in a trial evaluating risdiplam▼ in patients with spinal muscular atrophy (SMA) who experienced a plateau or decline in function after gene therapy
K Gorni, A Hahn, K Kotulska-Jozwiak, E Keto, Y Li, CJ Guittari
Neuroscience | Spinal Muscular Atrophy

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Duration 60mins Vienna, Austria
Scientific Poster: RAINBOWFISH: 3-year efficacy and safety data of risdiplam▼ in infants with presymptomatic spinal muscular atrophy (SMA)
M Mazurkiewicz-Bełdzińska, RS Finkel, L Servais, MA Farrar, D Vlodavets, E Zanoteli, M Al-Muhaizea, APQC Araújo, Leslie Nelson, Manni Kuthiala, K Gorni, H Kletzl, L Palfreeman, E Gaki, M Rabbia, D Summers, M Manfrini, E Bertini, on behalf of the RAINBOWFISH Study Group
Neuroscience | Spinal Muscular Atrophy

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Duration 60mins Vienna, Austria
5-year safety update: Risdiplam▼ clinical trial programme for spinal muscular atrophy (SMA)
R Masson, G Baranello, L Servais, BT Darras, JW Day, N Deconinck, MA Farrar, RS Finkel, E Bertini, J Kirschner, M Mazurkiewicz-Bełdzińska, S Bader-Weder, K Gorni, V Schlegel, Y Li, RS Scalco, E Mercuri, on behalf of the FIREFISH, SUNFISH, JEWELFISH and RAINBOWFISH Study Groups
Neuroscience | Spinal Muscular Atrophy

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Duration 60mins Vienna, Austria
Delandistrogene Moxeparvovec Micro-Dystrophin Expression and Safety in 3–4-year-olds with Duchenne Muscular Dystrophy in ENDEAVOR and ENVOL Studies
Jerry R. Mendell, Eugenio M. Mercuri, Andrea Gangfuss, Andrés Nascimento, Craig M. McDonald, Isabelle Desguerre, Craig M. Zaidman, Laurent Servais, Patsy Thrasher, Mark Vivien, Kai Ding, Alexander P. Murphy, Becca B. Zhang, Kathrin Wonde, Pooneh Pordeli, Louise R. Rodino-Klapac
Neuroscience | Duchenne Muscular Dystrophy

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01:30 PM
Duration 30mins Vienna
Oral Presentation: RAINBOWFISH: 3-year efficacy and safety data of risdiplam▼ in infants with presymptomatic spinal muscular atrophy (SMA)
M Mazurkiewicz-Bełdzińska, RS Finkel, L Servais, MA Farrar, D Vlodavets, E Zanoteli, M Al-Muhaizea, APQC Araújo, Leslie Nelson, Manni Kuthiala, K Gorni, H Kletzl, L Palfreeman, E Gaki, M Rabbia, D Summers, M Manfrini, E Bertini, on behalf of the RAINBOWFISH Study Group
Neuroscience | Spinal Muscular Atrophy

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01:45 PM
Duration 60mins Vienna, Austria
Retrospective assessment of feeding and nutrition after 2 years of risdiplam▼ treatment in younger children with spinal muscular atrophy (SMA) using the CEDAS
K Gorni, G Baranello, E Conway, Y Li
Neuroscience | Spinal Muscular Atrophy

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Duration 60mins Vienna, Austria
Delphi consensus project to determine the meaningful treatment outcomes in adult spinal muscular atrophy (SMA)
JF Vázquez-Costa, CA Chiriboga, T Duong, Y Erbas, J Glascock, N Gusset, R Muni-Lofra, C O’Connell, MC Walter, CJ Guittari, J Cochrane, LD Townson, D Riley, C Gasser, K Sully
Neuroscience | Spinal Muscular Atrophy

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Duration 60mins Vienna, Austria
A systematic literature review: Real-world motor function outcomes with risdiplam▼ in treatment-naïve adults with Types 2 and 3 SMA
K Gorni, CS Sutherland, E Davies, A Mahajan, S Kumari, A Simpson
Neuroscience | Spinal Muscular Atrophy

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Duration 60mins Vienna, Austria
Delandistrogene moxeparvovec in Duchenne muscular dystrophy: Long-term EMBARK 2-year functional outcomes, safety, and micro-dystrophin expression
Jerry R. Mendell, Francesco Muntoni, Craig M. McDonald, Eugenio M. Mercuri, Emma Ciafaloni, Hirofumi Komaki, Carmen Leon-Astudillo, Andrés Nascimento, Crystal Proud, Ulrike Schara-Schmidt, Aravindhan Veerapandiyan, Craig M. Zaidman, Alexander P. Murphy, Louise R. Rodino-Klapac, on behalf of the EMBARK Study Group
Neuroscience | Duchenne Muscular Dystrophy

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06:00 AM
Duration 30mins Hall D
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; Lilly East; Alexander P. Murphy; Christoph Wandel; James Richardson
Neuroscience | Duchenne Muscular Dystrophy

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10:45 AM
Duration 30mins Hall D
Acute Liver Injury Mitigation and Management in Patients with Duchenne Muscular Dystrophy Following Administration of Delandistrogene Moxeparvovec: Expert Committee Considerations
Perry Shieh, Crystal Proud, Tamir Diamond, Catherine Chapin, Jawad Ahmad, Alan Salama, Carsten Bönnemann, Jonathan Soslow, Barry J. Byrne, Aravindhan Veerapandiyan, John Brandsema, Susan Matesanz, Diana Bharucha-Goebel, Benjamin Samelson-Jones, Benjamin Wilkins, Marianne Gerber, Stefanie Mason, Damon Asher, Craig McDonald
Neuroscience | Duchenne Muscular Dystrophy

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