FIREFISH Parts 1 and 2: 4-year efficacy and safety of risdiplam in Type 1 spinal muscular atrophy (SMA) Mazurkiewicz-Beldzinska, M., Baranello, G., Boespflug-Tanguy, O., Day, J., Deconinck, N., Klein, A., Masson, R., Mercuri, E., Rose, K., Servais, L., Vlodavets, D., Xiong, H., Zanoteli, E., El-Khairi, M., Gerber, M., Gorni, K., Kletzl, H., Palfreeman, L., Dodman, A., Darras, B. WILEY. 2023: 196

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