FP.24 RAINBOWFISH: Preliminary efficacy and safety data in risdiplam-treated infants with presymptomatic spinal muscular atrophy (SMA)
Titel:
FP.24 RAINBOWFISH: Preliminary efficacy and safety data in risdiplam-treated infants with presymptomatic spinal muscular atrophy (SMA)
Auteur:
Finkel, R. Farrar, M. Vlodavets, D. Zanoteli, E. Al-Muhaizea, M. Nelson, L. Prufer, A. Servais, L. Wang, Y. Fisher, C. Gerber, M. Gorni, K. Kletzl, H. Palfreeman, L. Scalco, R. Bertini, E.