Result: Outcomes for patients in the RESTORE registry with spinal muscular atrophy and four or more SMN2 gene copies treated with onasemnogene abeparvovec

*Title*:

*Author/editor-in-chief*:

*Publication*:

Freiburg : Universität, 2025

*Physical description scale*:

1 Online-Ressource

*Format*:

*eBook*

*Language*:

*eng*

*Notes*:

European journal of paediatric neurology. - 53 (2024) , 18-24, ISSN: 1532-2130

*DOI*:

10.1016/j.ejpn.2024.08.006

*Result*: Outcomes for patients in the RESTORE registry with spinal muscular atrophy and four or more SMN2 gene copies treated with onasemnogene abeparvovec