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WMS 2023

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
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
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
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
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
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
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
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
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
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
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
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