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Roche and Genentech at

WMS 2025

We will be contributing to topics related to
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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
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, 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
Duration 60mins Vienna, Austria
Safety update: Risdiplam▼ clinical trial program 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
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
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
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
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
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
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
Coming soon