Poster 212 : RAINBOWFISH: Primary efficacy and safety data in risdiplam-treated infants with presymptomatic spinal muscular atrophy (SMA)

Finkel R, Farrar M, Servais L, Vlodavets D, Zanoteli E, Al-Muhaizea M, Prufer A, Nelson L, Fischer C, Gerber M, Gorni K, Kletzl H, Palfreeman L, Gaki E, Fontoura P, Bertini E, RAINBOWFISH Study Grp

Neuromuscular disorders : NMD