Nusinersen in infants who initiate treatment in a presymptomatic stage of spinal muscular atrophy (SMA): Interim results from the phase 2 nurture study

MM Ryan, DC De Vivo, E Bertini, WL Hwu, TO Crawford, KJ Swoboda, RS Finkel, J Kirschner, NL Kuntz, J Parsons, RJ Butterfield, H Topaloglu, T Ben Omran, VA Sansone, YJ Jong, F Shu, R Foster, I Bhan, S Fradette, W Farwell