1. Review of delandistrogene moxeparvovec in Duchenne muscular dystrophy patients.
2. Limitations in demonstrated clinical benefit despite FDA approval.
3. Minimal improvements in secondary motor outcomes.
4. Safety concerns include immune-related side effects, thrombocytopenia, and liver toxicity.
5. Limitations in using expression of microdystrophin as a surrogate endpoint.

Source: Neurology