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Home Neuroscience WMS 2023
Roche and Genentech at

WMS 2023

October 03 - October 07
Charleston, USA
wms2023.com
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Medical Materials
Agenda
Coming soon
02:30 PM
Duration 1hr Charleston, USA
Safety update: Risdiplam▼ clinical trial program for spinal muscular atrophy (SMA)
G Baranello, CA Chiriboga, L Servais, BT Darras, JW Day, NDeconinck, MA Farrar, RS Finkel, E Bertini, J Kirschner, R Masson, M Mazurkiewicz-Bełdzińska, D Vlodavets, S Bader-Weder, K Gorni, B Jaber, T McIver, G Papp, RS Scalco, E Mercuri
Neuroscience | Spinal Muscular Atrophy

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Duration 1hr Charleston, USA
Bioavailability and bioequivalence of risdiplam▼ tablets in healthy volunteers
H Kletzl, K Heinig, B Jaber, B Lomeli, WY Yeung, H Coleman, D Morrison
Neuroscience | Spinal Muscular Atrophy

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Duration 1hr Charleston, USA
MANATEE: GYM329 (RO7204239) in combination with risdiplam▼ treatment in patients with spinal muscular atrophy (SMA)
T Duong, BT Darras, J Morrow, F Muntoni, L Servais, M Rabbia, M Gerber, H Kletzl, E Gaki, S Fletcher, RS Scalco, K Wagner, E Mercuri
Neuroscience | Spinal Muscular Atrophy

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Duration 1hr Charleston, USA
EMBARK, a Phase 3 trial evaluating safety and efficacy of delandistrogene moxeparvovec in DMD: Study design and baseline characteristics
Muntoni F, Mercuri E, Schara-Schmidt U, Komaki H, Richardson J,Singh T, Guridi M, Mason S, Murphy AP, Yu L, Reid C, Darton E, Wandel C, Mendell JR
Neuroscience | Duchenne Muscular Dystrophy

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Duration 1hr Charleston, USA
ENVISION, a Phase 3, randomized trial evaluating the safety and efficacy of delandistrogene moxeparvovec in Duchenne muscular dystrophy: Study design
F Muntoni, E Mercuri, C McDonald, I Desguerre, Ma Tulinius, C Proud, M Furgerson, A P. Murphy, C De Ford, T Feng, C Reid, C Wandel, N Shelton
Neuroscience | Duchenne Muscular Dystrophy

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Duration 1hr Charleston, USA
ENVOL, a Phase 2, open-label trial evaluating the safety and expression of delandistrogene moxeparvovec in Duchenne muscular dystrophy: Study design
E Mercuri, I Desguerre, A Gangfuss, L Servais, A Nascimento, B B Zhang, A P. Murphy, C Reid, C Wandel, Teji Singh, M Guridi, F Muntoni
Neuroscience | Duchenne Muscular Dystrophy

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Duration 1hr Charleston, USA
Practical considerations for delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy
JR Mendell, C Proud, C Zaidman, S Mason, E Darton, C Wandel, A Murphy, E Mercuri, F Muntoni, C McDonald
Neuroscience | Duchenne Muscular Dystrophy

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Duration 1hr Charleston, USA
T-cell response to micro-dystrophin in a patient treated with delandistrogene moxeparvovec gene therapy: A case of immune-mediated myositis
S Khan, H Haegal, A Holleinstein, C Wandel, D Asher, D Griffin, R Potter, I Moeller, T Singh, L Rodino-Klapac
Neuroscience | Duchenne Muscular Dystrophy

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03:30 PM
Duration 30mins Ballroom C3
RAINBOWFISH: Primary efficacy and safety data in risdiplam▼-treated infants with presymptomatic spinal muscular atrophy SMA
RS Finkel, MA Farrar, L Servais, D Vlodavets, E Zanoteli, M Al-Muhaizea, A Prufer, 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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03:30 PM
Duration 1hr Charleston, USA
Long-term comparative efficacy and safety of risdiplam▼ versus nusinersen in children with Type 1 SMA
N Hawkins, R Evans, G Sajeev, A Mahajan, DA Scott, J Nam, CS Sutherland, C Kokaliaris
Neuroscience | Spinal Muscular Atrophy

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Duration 1hr Charleston, USA
Real-world treatment with risdiplam▼ in adults with SMA: A multicenter study
CJ Guittari, S Candrilli, LS Miles, A Simpson, S Shapouri
Neuroscience | Spinal Muscular Atrophy

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Duration 1hr Charleston, USA
Risdiplam▼ experience following onasemnogene abeparvovec in individuals with spinal muscular atrophy: a multicenter case series
Kuntz NL, Svoboda MD, Leon-Astudillo C, Byrne B, Krueger J, Kwon JM, Sieburg C, Castro D
Neuroscience | Spinal Muscular Atrophy

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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.