P.349Onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy Type 1 (SMA1): Pivotal phase 3 study (STR1VE) update
Title:
P.349Onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy Type 1 (SMA1): Pivotal phase 3 study (STR1VE) update
Author:
Day, J. Chiriboga, C. Crawford, T. Darras, B. Finkel, R. Connolly, A. Iannaccone, S. Kuntz, N. Pena, L. Schultz, M. Shieh, P. Smith, E. Feltner, D. Ogrinc, F. Ouyang, H. Macek, T. Kernbauer, E. Sproule, D. Authorship Truncated, Mendell, J.