Answer
It targets SMN2 splicing, which can partly compensate for the faulty SMN1 gene responsible for 5q SMA. It is not established for unrelated causes of motor-neuron weakness.
Sources for this answer
- Evrysdi 0.75 mg/mL powder for oral solutionemc · SmPC Current selected SmPC · Version 12
- Evrysdi 5 mg film‑coated tabletsemc · SmPC Current selected SmPC · Version 1