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Results 1-4 of 4 (Search time: 0.027 seconds).
Issue DateTitleAuthor(s)
2021IREFISH Parts 1 and 2: 24-month safety and efficacy of risdiplam in type 1 spinal muscular atrophy (SMA)MASSON, R.; BOESPFLUG-TANGUY, O.; DARRAS, B.; DAY, J.; DECONINCK, N.; KLEIN, A.; MAZURKIEWICZ-BELDZINSKA, M.; MERCURI, E.; ROSE, K.; SERVAIS, L.; VLODAVETS, D.; XIONG, H.; ZANOTELI, E.; DODMAN, A.; EL-KHAIRI, M.; GAKI, E.; GERBER, M.; GORNI, K.; KLETZL, H.; BARANELLO, G.
2021RAINBOWFISH: A study of risdiplam in infants with presymptomatic spinal muscular atrophy (SMA)SERVAIS, L.; AL-MUHAIZEA, M.; FARRAR, M.; NELSON, L.; PRUFER, A.; FINKEL, R.; WANG, Y.; ZANOTELI, E.; EL-KHAIRI, M.; GERBER, M.; GORNI, K.; KLETZL, H.; PALFREEMAN, L.; SCALCO, R.; BERTINI, E.
2021ANCHOVY: A retrospective cohort study of the natural history of type 1 spinal muscular atrophy (SMA) using medical record dataZANOTELI, E.; CANCES, C.; VLODAVETS, D.; COMI, G.; MASSON, R.; MAZURKIEWICZ-BELDZINSKA, M.; SAITO, K.; DODMAN, A.; EL-KHAIRI, M.; GORNI, K.; GRAVESTOCK, I.; HOFFART, J.; SCALCO, R.; DARRAS, B.
2021Clinical and genetic spectrum of a large cohort of delta-sarcoglycan muscular dystrophyALONSO-PEREZ, J.; GONZALEZ-QUEREDA, L.; BRUNO, C.; PANICUCCI, C.; ALAVI, A.; ZANOTELI, E.; MUELAS, N.; VILCHEZ, J.; DOURADO, E.; KADEM, N.; UMAIR, M.; GUGLIERIR, M.; MARINI-BETTOLO, C.; STRAUB, V.; DIAZ-MANERA, J.
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