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Home Neuroscience CURE SMA 2023
Roche and Genentech at

CURE SMA 2023

June 29 - July 02
Orlando, USA
curesma.org
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Medical Materials
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Jun 30 / Roche and Genentech
SUNFISH Parts 1 and 2: 4-year efficacy and safety of risdiplam▼ in Types 2 and 3 SMA
SUNFISH (NCT02908685) is a multicenter, two-part, randomized (2:1, risdiplam▼:placebo), placebo-controlled, double-blind study evaluating the efficacy and safety of risdiplam▼ in patients with Type 2 or Type 3 SMA (inclusion criteria: 2–25 years at enrollment). This presentation will report safety and efficacy data from Parts 1 and 2 of the SUNFISH study after 4 years of treatment with risdiplam▼.
Neuroscience Spinal Muscular Atrophy CURE-SMA-2023

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Jun 30 / Roche and Genentech
FIREFISH Parts 1 and 2: 48-month safety and efficacy of risdiplam▼ in Type 1 SMA
FIREFISH (NCT02913482) is an ongoing, multicenter, open-label study of risdiplam▼ in infants aged 1–7 months with Type 1 SMA and two SMN2 gene copies. Part 1 assessed the safety, tolerability, PK and PD of different risdiplam▼ dose levels; Part 2 assesses the safety and efficacy of the Part 1-selected dose of risdiplam▼. This presentation will report data from children in Part 1 and 2 who have received risdiplam▼ treatment for 48 months.
Neuroscience Spinal Muscular Atrophy CURE-SMA-2023

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Jun 29 / Roche and Genentech
RISDIPLAM▼ EXPERIENCE FOLLOWING ONASEMNOGENE ABEPARVOVEC IN INDIVIDUALS WITH SPINAL MUSCULAR ATROPHY: A MULTICENTER CASE SERIES
Limited data are available on the combination of currently approved SMA therapies and additional evidence is needed. This presentation reports on a multicenter retrospective review of children with SMA who were treated with onasemnogene abeparvovec and subsequently risdiplam▼ in the US. Demographics, clinical characteristics, adverse events, hospitalizations and outcomes are described for each case as applicable. These results aim to increase understanding of the risk-benefit profile for the combined use of risdiplam▼ after onasemonogene abeparvovec.
Neuroscience Spinal Muscular Atrophy CURE-SMA-2023
02:40 PM
Duration 20mins Walt Disney World Swan Hotel
RISDIPLAM▼ EXPERIENCE FOLLOWING ONASEMNOGENE ABEPARVOVEC IN INDIVIDUALS WITH SPINAL MUSCULAR ATROPHY: A MULTICENTER CASE SERIES
Melissa Svoboda, Carmen Leon Astudillo, Barry Byrne, Jena Krueger, Nancy Kuntz, Jennifer Kwon, Cory Seiburg, Diana Castro
Neuroscience | Spinal Muscular Atrophy

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10:00 AM
Duration 20mins Walt Disney World Swan Hotel
FIREFISH Parts 1 and 2: 48-month safety and efficacy of risdiplam▼ in Type 1 SMA
G Baranello, M Mazurkiewicz-Bełdzińska, O Boespflug-Tanguy, JW Day, N Deconinck, A Klein, R Masson, E Mercuri, K Rose, L Servais, D Vlodavets, H Xiong, E Zanoteli, M El-Khairi, M Gerber, K Gorni, H Kletzl, L Palfreeman, A Dodman, BT Darras
Neuroscience | Spinal Muscular Atrophy

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10:20 AM
Duration 20mins Walt Disney World Swan Hotel
SUNFISH Parts 1 and 2: 4-year efficacy and safety of risdiplam▼ in Types 2 and 3 SMA
JW Day, N Deconinck, ES Mazzone, A Nascimento, M Oskoui, K Saito, C Vuillerot, G Baranello, O Boespflug-Tanguy, N Goemans, J Kirschner, A Kostera-Pruszczyk, L Servais, J Braid, G Papp, K Gorni, C Martin, WY Yeung, E Mercuri
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.