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Home Neuroscience Cure SMA 2025
Roche and Genentech at

Cure SMA 2025

June 25 - June 27
Anaheim, USA
curesma.org
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Medical Materials
Agenda
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11:30 PM
Duration 120mins Anaheim, USA
Baseline demographics, clinical characteristics and treatment patterns of risdiplam▼-treated individuals with SMA from the (PNCR) registry
B T Darras, S Roumpanis, W Martens, S Shapouri, T Dickendesher, C J Guittari, J W Day, R S Finkel, M Hirano, D Jayaraman, Z Zolkipli-Cunningham, S Edwards
Neuroscience | Spinal Muscular Atrophy

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Duration 120mins Anaheim, USA
Real-world risdiplam▼ effectiveness in adults with SMA from the Pediatric Neuromuscular Clinical Research (PNCR) registry
D Jayaraman, S Roumpanis, W Martens, T Dickendesher, S Shapouri, B T Darras, M Hirano,Z Zolkipli-Cunningham, A Gershon
Neuroscience | Spinal Muscular Atrophy

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Duration 120mins Anaheim, USA
Delphi consensus project to determine the meaningful treatment outcomes in adult SMA
K Sully, CA Chiriboga, T Duong, Y Erbas, J Glascock, N Gusset, R Muni-Lofra, C O'Connell, JF Vázquez-Costa, MC Walter, CJ Guittari, J Cochrane,LD Townson, A Marciniak, D Riley, C Gasser
Neuroscience | Spinal Muscular Atrophy

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Duration 120mins Anaheim, USA
Clinical outcomes among risdiplam▼-treated patients with spinal muscular atrophy (SMA) in the Cure SMA Clinical Data Registry
S Shapouri, TM To, S Whitmire, L Belter, M Schroth, TL Dickendesher, S Tam, R Schuldt
Neuroscience | Spinal Muscular Atrophy

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Duration 120mins Anaheim, USA
Examination of outcomes data for risdiplam▼-treated individuals with SMA using the Cure SMA Community Update Survey
TM To, R Schuldt,TL Dickendesher, S Tam, S Shapouri
Neuroscience | Spinal Muscular Atrophy

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08:00 PM
Duration 120mins Anaheim, USA
International evidence-based consensus on the management of gastrointestinal adverse events related to the use of risdiplam▼ in patients with spinal muscular atrophy
Manisha K Korb and Channa Hewamadduma on behalf of the SMA Delphi Panel
Neuroscience | Spinal Muscular Atrophy

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Duration 120mins Anaheim, USA
Defining the clinical benefits of disease-modifying therapy in people living with spinal muscular atrophy who are permanently ventilated: a UK-based Delphi consensus process
Mariacristina Scoto on behalf of the SMA Delphi Panel
Neuroscience | Spinal Muscular Atrophy

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Duration 120mins Anaheim, USA
Real-world use of risdiplam▼ for the treatment of SMA in infants under 2 months of age
N Goedeker, VJ Carson, S Bernes, CC Haws, C Zaidman, C Campos, I Yermilov, MS Broder, E Chang, S Edwards, TL Dickendesher, S Tam, S Shapouri
Neuroscience | Spinal Muscular Atrophy

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Duration 120mins Anaheim, USA
A long-term follow-up study of risdiplam▼ (WeSMA study): Interim results in adult participants with SMA
Abdul Hamid, K Arya, S Bernes, JL Chinarian, CA Chiriboga, AK Daunter, S Dixon, A Kumar, S Ladha, SE Matesanz, LM Ramos-Platt, RC Richardson, ​RJ Scharf, MD Svoboda, DN Toupin, AS Varadhachary, V Vedanarayanan, SE Wright, I Tanvir, E Lim, E Keto, S Shapouri, R Shah, G Seth​
Neuroscience | Spinal Muscular Atrophy

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10:20 PM
Duration 20mins Disney's Grand Californian Hotel
Retrospective assessment of feeding and nutrition after 2 years of risdiplam▼ treatment in younger children with SMA using the CEDAS
G Baranello, E Conway ,Y Li, K Gorni
Neuroscience | Spinal Muscular Atrophy

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11:00 PM
Duration 120mins Anaheim, USA
The comparative efficacy and safety of risdiplam▼ versus high-dose nusinersen in children with Type 1 SMA
C Gasser, C Kokaliaris, N Hawkins, A Mahajan, D Alexander Scott, C. E Sideris
Neuroscience | Spinal Muscular Atrophy

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Duration 120mins Anaheim, 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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