Quijano-Roy, S., Parsons, J., Aharoni, S., Lakhotia, A., TIZZANO, E. F., & Servais, L. (2024). Outcomes for patients in the RESTORE registry with spinal muscular atrophy and four or more SMN2 gene copies treated with onasemnogene abeparvovec.

Quijano-Roy, Susana, Julie Parsons, Sharon Aharoni, Arpita Lakhotia, EDUARDO F. TIZZANO, and Laurent Servais. Outcomes for Patients in the RESTORE Registry With Spinal Muscular Atrophy and Four or More SMN2 Gene Copies Treated With Onasemnogene Abeparvovec. 2024.

Quijano-Roy, Susana, et al. Outcomes for Patients in the RESTORE Registry With Spinal Muscular Atrophy and Four or More SMN2 Gene Copies Treated With Onasemnogene Abeparvovec. 2024.