RAINBOWFISH: a study of risdiplam (RG7916) in infants with pre-symptomatic spinal muscular atrophy (SMA) Bertini, E., Day, J. W., Al Muhaizea, M., Xiong, H., Servais, L., Prufer, A., Buchbjerg, J., Yeung, W. Y., Gorni, K., Khwaja, O. WILEY. 2019: 445

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