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Cure SMA 2023

June 29 - July 02

Orlando, USA

curesma.org

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Medical Materials

Agenda

Coming soon

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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