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SMA Europe 2026

We will be contributing to topics related to
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03:15 PM
Duration 75mins Budapest, Hungary
The comparative efficacy and safety of risdiplam versus high-dose nusinersen in children with Type 1 SMA
Eleftherios Sideris, Cyrill Gasser, Christos Kokaliaris, Neil Hawkins, Anadi Mahajan, David Alexander Scott
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Duration 75mins Budapest, Hungary
HINALEA 2: Baseline observations in a trial evaluating risdiplam in patients with SMA who experienced a plateau or decline in function after gene therapy
Katarzyna Kotulska-Jozwiak, Carol Jean Guittari, Prof Andreas Hahn, Erica Keto, Yue Li, Ksenija Gorni
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Duration 75mins Budapest, Hungary
Defining the clinical benefits of disease-modifying therapy in people living with spinal muscular atrophy who are permanently ventilated: a UK-based Delphi consensus process
Mariacristina Scoto
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03:15 PM
Duration 75mins Budapest, Hungary
Real-world motor function outcomes with risdiplam in Types 2 and 3 SMA: A systematic literature review and meta-analysis
Eleftherios Sideris, Simone Sutherland, Evan Davies, Ksenija Gorni, Susan Edwards, CJ Guittari, Anadi Mahajan, Sandhya Kumari, Eletherios Sideris, Maya Kjaergaard, Digant Gupta, Mudasir Khan
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Duration 75mins Budapest, Hungary
Final results from the JEWELFISH study: 5-year risdiplam treatment in non-treatment-naïve individuals with SMA
Claudia Chiriboga, Claudio Bruno, Tina Duong, Dirk Fischer, Janbernd Kirschner, Mariacristina Scoto, Eugenio Mercuri, Valerie Schlegel, Ksenija Gorni, Heidemarie Kletzl, Pooneh Pordelli, Eleni Gaki, Francis Warren, Francesco Muntoni
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Duration 75mins Budapest, Hungary
RAINBOWFISH: 3-year efficacy and safety data of risdiplam in children with presymptomatic SMA
Maria Mazurkiewicz-Bełdzińska, Richard Finkel, Laurent Servais, Michelle Farrar, Dmitry Vlodavets, Edmar Zanoteli, Mohammad Al Muhaizea, Alexandra Araujo, Leslie Nelson, Manni Kuthiala, Ksenija Gorni, Heidemarie Kletzl, Francis Warren, Eleni Gaki, Dave Summers, Marianna Manfrini, Enrico Bertini
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Duration 75mins Budapest, Hungary
5-year safety update: Risdiplam clinical trial program for SMA
Mariacristina Scoto, Riccardo Masson, Giovanni Baranello, Laurent Servais, Basil Darras, John Day, Nicolas Deconinck, Michelle Farrar, Richard Finkel, Enrico Bertini, Janbernd Kirschner, Maria Mazurkiewicz-Beldzinksa, Silvia Bader-Weder, Ksenija Gorni, Valerie Schlegel, Yue Li , Renata Scalco, Eugenio Mercuri
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Duration 75mins Budapest, Hungary
International evidence-based consensus on the management of gastrointestinal adverse events related to the use of risdiplam in patients with SMA
Channa Hewamadduma, Manisha Korb
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