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MDA 2026

We will be contributing to topics related to
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10:00 PM
Duration 120mins Orlando, USA
RAINBOWFISH: 3-year efficacy and safety data of risdiplam in children with presymptomatic (SMA)
Laurent Servais
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Duration 120mins Orlando, USA
5-year safety update: Risdiplam clinical trial program for spinal muscular atrophy (SMA)
Carol Jean Guittari
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Duration 120mins Orlando, USA
The comparative efficacy and safety of risdiplam versus high-dose nusinersen in children with Type 1 spinal muscular atrophy (SMA)
Julian Nam
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Duration 120mins Orlando, USA
Caregiver Global Impressions of Delandistrogene Moxeparvovec in Participants with Duchenne Muscular Dystrophy: Findings from EMBARK 2-year Follow-up
Teofil Ciobanu
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Duration 120mins Orlando, USA
Pooled Safety Analysis from Phase 1 to Phase 3 Clinical Trials of Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy
Craig M. McDonald
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Duration 120mins Orlando, USA
Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy: Functional and Safety Outcomes up to 3 Years Post-Infusion in the EMBARK Study
Craig M. McDonald
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10:00 PM
Duration 120mins Orlando, USA
Fertility in Risdiplam-Treated Adult Males with Spinal Muscular Atrophy (SMA): the MARLIN study
Sheila Shapouri, Sheila Seleri, Natan Bar-Chama, Shafeeq Ladha, Shereen Yap, Elise Lim, Rushabh Shah, Travis Dickendesher
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06:30 PM
Duration 15mins Hilton Orlando, Orlando, FL, Room Name: Florida 4
Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy: Functional and Safety Outcomes up to 3 Years Post-Infusion in the EMBARK Study
Craig M. McDonald
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