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ICNMD 2022

We will be contributing to topics related to
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04:15 PM
Duration 15mins Brussels, Belgium/ Virtual (Hybrid)
Phase 1/2 trial of delandistrogene moxeparvovec in patients with DMD: 4-year update
Jerry R. Mendell, Zarife Sahenk, Kelly J. Lehman, Carrie Nease, Linda P. Lowes, Natalie F. Reash, Megan A. Iammarino, Lindsay N. Alfano, Jordan Vaiea, Sarah Lewis, Kathleen Church, Richard Shell, Rachael A. Potter, Danielle A. Griffin, Eric R. Pozsgai, Mark Hogan, Larry Hu, Kathryn Giblin, Louise R. Rodino-Klapac
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04:15 PM
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SUNFISH: 3-year efficacy and safety of risdiplam▼ in Types 2 and 3 spinal muscular atrophy
Nicolas Deconinck, John W Day, Elena S Mazzone, Andres Nascimento, Maryam Oskoui, Kayoko Saito, ​Carole Vuillerot, Giovanni Baranello, Odile Boespflug-Tanguy, Nathalie Goemans, Janbernd Kirschner, Anna Kostera-Pruszczyk, Laurent Servais, Jessica Braid, Marianne Gerber, Ksenija Gorni, Carmen Martin, Renata S Scalco, Wai Yin Yeung, Eugenio Mercuri, on behalf of the SUNFISH Working Group
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05:00 PM
Duration 15mins Brussels, Belgium/ Virtual (Hybrid)
RAINBOWFISH: Preliminary efficacy and safety data in risdiplam▼-treated infants with presymptomatic 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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05:15 PM
Duration 15mins Brussels, Belgium/ Virtual (Hybrid)
Risdiplam▼: pharmacokinetic, pharmacodynamic, safety and efficacy exposure response analyses ​
Heidemarie Kletzl, Yumi Cleary, Paul Grimsey, Marianne Gerber, Renata S Scalco
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05:30 PM
Duration 15mins Brussels, Belgium/ Virtual (Hybrid)
Matching-adjusted Indirect Comparison of Risdiplam▼ Versus Nusinersen in Type 1 Spinal Muscular Atrophy: 2-year Update​
Neil Hawkins, Valerie Aponte Ribero, Ksenija Gorni, Monica Daigl, Yasmina Martí, Rachel Evans, David Alexander Scott, Anadi Mahajan ​
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