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Home Neuroscience Cure SMA 2026
Roche and Genentech at

Cure SMA 2026

June 24 - June 26
Florida, USA
curesma.org
We will be contributing to topics related to
Neuroscience
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Agenda
Medical Materials
11:30 AM
Duration 120mins Florida, USA
HINALEA 2: Baseline observations in a trial evaluating risdiplam in patients with SMA who experienced a plateau or decline in function after gene therapy
CJ Guittari, A Hahn, K Kotulska-Jozwiak, E Keto, K Gorni, Y Li
Neuroscience | Spinal Muscular Atrophy

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Duration 120mins Florida, USA
Patient- and caregiver-reported swallowing and feeding outcomes in individuals with SMA treated with risdiplam: A longitudinal analysis of the Cure SMA Community Update Survey
Sheila Shapouri, Robert Schuldt, Lisa Belter, Mary Curry, Helen Lin, Ksenija Gorni
Neuroscience | Spinal Muscular Atrophy

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11:00 AM
Duration 120mins Florida, USA
A long-term follow-up study of risdiplam (WeSMA study): 12-month results in adult participants with SMA
Sheila Seleri, Elise Lim, Erica Keto, Rushabh Shah, Sheila Shapouri
Neuroscience | Spinal Muscular Atrophy

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Duration 120mins Florida, USA
Fertility in Risdiplam-Treated Adult Males with Spinal Muscular Atrophy (SMA):
Sheila Shapouri, Sheila Seleri, Natan Bar-Chama, Shafeeq Ladha, Shereen Yap, Elise Lim, Rushabh Shah, Travis Dickendesher
Neuroscience | Spinal Muscular Atrophy

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Duration 120mins Florida, USA
Evaluating the Feasibility of Linking Clinical Trial Data to Real-World Data Using Tokenization in Spinal Muscular Atrophy: Insights from the WeSMA Trial
Rekha Shivapa, Abigail Alve, Sheila Seleri, Karen Sick, Kimberly Barnholt, Erica Keto, Thomas Vaccaro
Neuroscience | Spinal Muscular Atrophy

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04:10 PM
Duration 20mins Florida
Safety and pharmacodynamic effects of emugrobart in combination with risdiplam in ambulant and non-ambulant participants with SMA: MANATEE Part 1
L Servais, B T Darras, E Mercuri, J M Morrow, F Muntoni, R Abbas, C De Ford, S Fletcher, E Gaki, H Kletzl, M Kuthiala, R S Scalco, V Schlegel, K R Wagner, W Yeung, T Duong
Neuroscience | Spinal Muscular Atrophy

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04:30 PM
Duration 20mins Florida
Final results from the JEWELFISH study: 5-year risdiplam treatment in non-treatment-naïve individuals with SMA
C A Chiriboga, C Bruno, T Duong, D Fischer, M Scoto, E Mercuri,V Schlegel, K Gorni, H Kletzl, P Pordeli, E Gaki, F Warren, F Muntoni
Neuroscience | Spinal Muscular Atrophy

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