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Home Neuroscience CURE SMA 2024
Roche and Genentech at

CURE SMA 2024

June 06 - June 09
Texas, USA
curesma.org
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Medical Materials
Agenda
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09:30 PM
Duration 2hrs Texas, USA
Fertility outcomes in risdiplam▼-treated male patients with SMA: A multicenter case series
Shelley Coskery, Marcus Erdler, Margaret R. Frey, Michael Lopez
Neuroscience | Spinal Muscular Atrophy

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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
Neuroscience | Spinal Muscular Atrophy

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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
Neuroscience | Spinal Muscular Atrophy

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Duration 2hrs Texas, USA
Post hoc analysis of the SMA Independence Scale-Upper Limb Module (SMAIS-ULM) in individuals with Type 2 and non-ambulant Type 3 SMA using SUNFISH Part 2 data
K Sully, L Gao, C J Guittari, J Braid
Neuroscience | Spinal Muscular Atrophy

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Duration 2hrs Texas, USA
Bioequivalence and food effect assessment for a new room-temperature risdiplam▼ tablet formulation in healthy volunteers
H Kletzl, K Heinig, B Jaber, W Y Yeung, H Coleman, B Lomeli, L Buckley, A Varghese
Neuroscience | Spinal Muscular Atrophy

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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
Neuroscience | Spinal Muscular Atrophy

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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
Neuroscience | Spinal Muscular Atrophy

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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
Neuroscience | Spinal Muscular Atrophy

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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
Neuroscience | Spinal Muscular Atrophy

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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
Neuroscience | Spinal Muscular Atrophy

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Duration 2hrs Texas, USA
Using privacy preserving record linkage to incorporate real-world data into a Phase IV long term follow-up of individuals with SMA (WeSMA)
A Alve, I Tanvir, R Shivapa, S Tam, K Gavin, M Witman, B Assman
Neuroscience | Spinal Muscular Atrophy

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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
Neuroscience | Spinal Muscular Atrophy

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