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Cure SMA 2021

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Coming soon
02:00 PM
Duration 15mins Virtual
JEWELFISH: Safety, pharmacodynamic and exploratory efficacy data in non-naïve patients with spinal muscular atrophy (SMA) receiving treatment with risdiplam▼
Chiriboga CA*, Bruno C, Duong T, Fischer D, Kirschner J, Mercuri E, Gerber M, Gorni K, Kletzl H, Carruthers I, Martin C, Warren F, Scoto M on behalf of the JEWELFISH Study Group

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Duration 15mins Virtual
RAINBOWFISH: A study of risdiplam▼ in infants with presymptomatic spinal muscular atrophy (SMA)
Finkel RS*, Al-Muhaizea M, Farrar MA, Nelson L, Prufer A, Servais L, Wang Y, Zanoteli E, El-Khairi M, Gerber M, Gorni K, Kletzl H, Palfreeman L, Scalco RS, Bertini E on behalf of the RAINBOWFISH Study Group

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