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MDA 2024

Coming soon
11:00 PM
Duration 2hrs Hilton Orlando, Florida
Bioequivalence and food effect assessment for a room-temperature stable risdiplam▼ tablet formulation in healthy volunteers
H Kletzl, K Heinig, B Jaber, W Y Yeung, H Coleman, B Lomeli, L Buckley, A Varghese
Duration 2hrs Hilton Orlando, Florida
Correlation of individual SMN protein with observed efficacy in the risdiplam▼ clinical trials
H Kletzl, Y Cleary, P Grimsey, CA Chiriboga, E Mercuri, T McIver, R Thomas, RS Scalco
Duration 2hrs Hilton Orlando, Florida
RAINBOWFISH: Primary efficacy and safety data in risdiplam▼-treated infants with presymptomatic SMA
RS Finkel, MA Farrar, L Servais, D Vlodavets, E Zanoteli, M Al-Muhaizea, APQC Araujo, L Nelson, B Jaber, K Gorni, H Kletzl, L Palfreeman, E Gaki, M Rabbia, D Summers, P Fontoura, E Bertini
Duration 2hrs Hilton Orlando, Florida
Real-World Outcomes of Infants who Initiate Risdiplam▼ Under 2 Months of Age for the Treatment of Spinal Muscular Atrophy (SMA) in the US
Sheila Shapouri, Dalia Moawad, Susan Edwards, Cynthia Campos, Irina Yermilov, Travis Dickendesher
Duration 2hrs Hilton Orlando, Florida
Risdiplam▼ experience following onasemnogene abeparvovec in individuals with spinal muscular atrophy (SMA): a multicenter case series
Nancy Kuntz, Melissa D Svoboda, C Leon-Astudillo, B Byrne, J Krueger, JM Kwon, C Sieburg, D Castro
Duration 2hrs Hilton Orlando, Florida
MARLIN: An Observational Study of Fertility in Risdiplam▼-Treated Adult Male Patients With Spinal Muscular Atrophy (SMA)
Travis Dickendesher, N Bar-Chama, S Ladha, T Nazem, S Yap, B Assman, E Lim, R Shah, S Shapouri
Duration 2hrs Hilton Orlando, Florida
Long-Term Follow-Up Study of Risdiplam▼ in Participants with Spinal Muscular Atrophy (WeSMA)
Imran Tanvir, E Lim, S Shapouri, R Shah, C Sun, G Seth
Duration 2hrs Hilton Orlando, Florida
Transgene-directed immunologic investigations into immune-mediated myositis following delandistrogene moxeparvovec▼ gene therapy
Stefanie Mason, Sohrab Khan, Helene Haegel, Andres Hollenstein,Christoph Wandel, Damon Asher, Danielle Griffin, Eddie Darton, Rachael Potter, Ida Moeller, Susan Iannaccone, Craig Zaidman, Louise Rodino-Klapac
03:45 PM
Duration 15mins Florida 1-3
Safety and efficacy of delandistrogene moxeparvovec versus placebo in Duchenne muscular dystrophy (EMBARK): Pivotal Phase 3 Primary results
Jerry R. Mendell, Francesco Muntoni, Craig M. McDonald, Eugenio M. Mercuri, Emma Ciafaloni, Hirofumi Komaki, Carmen Leon-Astudillo, Andrés Nascimento, Crystal Proud, Ulrike Schara-Schmidt, Aravindhan Veerapandiyan, Craig M. Zaidman, Maitea Guridi, Alexander P. Murphy, Carol Reid, Christoph Wandel, Damon R. Asher, Eddie Darton, Stefanie Mason, Rachael A. Potter, Teji Singh, Wenfei Zhang, Paulo Fontoura, Jacob S. Elkins, Louise R. Rodino-Klapac