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Coming soon
09:30 PM
Duration 2hrs Texas, USA
MARLIN: An Observational Study of Fertility in Adult Males with Spinal Muscular Atrophy Treated with Risdiplam▼
Travis Dickendesher, Natan Bar-Chama, Shafeeq Ladha, Taraneh Nazem, Shereen Yap, Beverly Assman, Elise Lim, Rushabh Shah, Sheila Shapouri
Duration 2hrs Texas, USA
Qualitative perspectives from individuals with SMA and caregivers regarding meaningful change on the Revised Hammersmith Scale in individuals (2-60 years of age) with SMA
K Sully, G Coratti, M Curry, Y Erbas, J Glascock, N Gusset, C Lilien, A Mayhew, R Arbuckle, J Flynn, V Smith, E Taylor, S Wallace, J Braid, K Zarzar
Duration 2hrs Texas, USA
Examination of Outcomes Data for Risdiplam▼-treated individuals with SMA using the Cure SMA Community Update Survey
Tu My To, Lisa Belter, Mary Curry, Mary Schroth, Travis Dickendesher, Sheila Shapouri
09:00 PM
Duration 2hrs Texas, USA
Real-world treatment with risdiplam▼ in adults with SMA: A multicenter study
Carol Jean Guittari, Sean Candrilli, LaStella Miles, Alex Simpson, Sheila Shapouri
Duration 2hrs Texas, USA
Correlation of individual survival of motor neuron (SMN) protein with observed efficacy in the risdiplam▼ clinical trials
H Kletzl, Y Cleary, P Grimsey, C A Chiriboga, E Mercuri, T McIver, R Thomas, R S Scalco
Duration 2hrs Texas, USA
Upper limb function: Experience from the risdiplam▼ clinical trial program
Sully K, Duong T, Mazzone E, Mercuri E, Gaki E, Gorni K, Guittari CJ, Li Y, Martin C, Yeung WY
Duration 2hrs Texas, USA
Long-term follow-up study of risdiplam▼ in participants with SMA (WeSMA)
Imran Tanvir, Elise Lim, Sheila Shapouri, Rushabh Shah, Christy Sun, Gaurav Seth
03:40 PM
Duration 20mins Austin Marriott Downtown
FIREFISH Parts 1 and 2: 5-year efficacy and safety of risdiplam▼ in Type 1 SMA
M Mazurkiewicz-Beldzinska, G Baranello, O Boespflug-Tanguy, J W Day, N Deconinck, A Klein, R Masson, E Mercuri, K Rose, L Servais, D Vlodavets, H Xiong, E Zanoteli, M El-Khairi, E Gaki, M Gerber, K Gorni, H Kletzl, L Palfreeman, B T Darras