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Cure SMA 2022

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Coming soon
10:40 AM
Duration 20mins The Sequoia ballroom (Grand Californian Hotel)
RAINBOWFISH: Preliminary efficacy and safety data in risdiplam▼-treated infants with presymptomatic spinal muscular atrophy (SMA)
Richard S Finkel, Michelle A Farrar, Dmitry Vlodavets, Edmar Zanoteli, Mohammad Al-Muhaizea, Leslie Nelson, Alexandra Prufer, Laurent Servais, Yi Wang, Carolyn Fisher, Marianne Gerber, Ksenija Gorni, Heidemarie Kletzl, Laura Palfreeman, Renata S Scalco, Enrico Bertini, on behalf of the RAINBOWFISH Study Group​

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11:00 AM
Duration 20mins The Sequoia ballroom (Grand Californian Hotel)
FIREFISH Parts 1 and 2: 36-month safety and efficacy of risdiplam▼ in Type 1 spinal muscular atrophy (SMA)​
Laurent Servais, Giovanni Baranello, Odile Boespflug-Tanguy, John W Day, Nicolas Deconinck, Andrea Klein, Riccardo Masson, Maria Mazurkiewicz-Beldzinska, Eugenio Mercuri, Kirsty Rose, Dmitry Vlodavets, Hui Xiong, Edmar Zanoteli, Muna El-Khairi, Marianne Gerber, Ksenija Gorni, Heidemarie Kletzl, Angela Dodman, Eleni Gaki, Basil T Darras, on behalf of the FIREFISH Working Group

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