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 solution — Current selected SmPCemc · Summary of Product Characteristics
- Evrysdi 5 mg film‑coated tablets — Current selected SmPCemc · Summary of Product Characteristics
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