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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
Coming soon
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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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.