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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
Coming soon
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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This website is intended for healthcare professionals outside the United Kingdom (UK) and Australia. Registration status and prescribing information of medicinal products may differ between countries. Please refer to local product information for any medicinal products mentioned on this website.