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FIREFISH Parts 1 and 2: 12-month pooled safety and efficacy outcomes of risdiplam (RG7916) in infants with Type 1 spinal muscular atrophy (SMA) Servais, L., Bloespflug-Tanguy, O., Darras, B., Day, J., Deconinck, N., Klein, A., Masson, R., Mazurkiewicz-Beldzinska, M., Mercuri, E., Rose, K., Vlodavets, D., Xiong, H., Zanoteli, E., Dodman, A., El-Khairi, M., Gerber, M., Gorni, K., Kletzl, H., Scalco, R., Baranello, G. PERGAMON-ELSEVIER SCIENCE LTD. 2020: S126
View details for DOI 10.1016/j.nmd.2020.08.270
View details for Web of Science ID 000579727800263