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ICNMD 2022

Coming soon
04:15 PM
Duration 15mins Meeting Studio 211 + 212
Phase 1/2a 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
04:15 PM
Duration 15mins Copper Hall
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
05:00 PM
Duration 15mins Copper Hall
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​
05:15 PM
Duration 15mins Copper Hall
Risdiplam▼: pharmacokinetic, pharmacodynamic, safety and efficacy exposure response analyses ​
Heidemarie Kletzl, Yumi Cleary, Paul Grimsey, Marianne Gerber, Renata S Scalco
05:30 PM
Duration 15mins Copper Hall
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 ​