Answer
It targets the underlying SMN1 deficiency but cannot restore motor neurons already lost; ongoing multidisciplinary SMA care remains necessary.
Sources for this answer
- Zolgensma 2 x 10Exp13 vector genomes/mL solution for infusion — Current selected SmPCemc · Summary of Product Characteristics
- NHSNHS · NHS medicine guidance
General medicine information. Follow the instructions for your own medicine and speak to a pharmacist or clinician about your individual circumstances.