Result: Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study

*Title*:

*Author/editor-in-chief*:

*Publication*:

Freiburg : Universität, 2024

*Physical description scale*:

1 Online-Ressource

*Format*:

*eBook*

*Language*:

*eng*

*Notes*:

The lancet. - 47 (2024) , 101092, ISSN: 2666-7762

*DOI*:

10.1016/j.lanepe.2024.101092

*Result*: Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study