Approved is not the same as reachable for every family tomorrow.

Indication language limits use to pediatric patients with neurologic manifestations and preserved developmental function. Children already deep into regression may not match the same approved window.

Centers must be ready for infusion reactions, steroid courses, and lab monitoring. Geography, insurance, and caregiver leave can make “approved” feel theoretical. Other rare brain diseases are not automatically covered by one approval.

First approval is real. Overclaim is the failure mode.