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Walk the Even Hospital Database by book and chapter — the raw source passages that ground Ask, DDx, and the rest.

1 passage

abstractpubmed· Abstract· item 35840728

Early treatment is a lifeline for infants with SMA. In the Phase 3 SPRINT trial, pre-symptomatic gene therapy demonstrated impressive clinical outcomes in infants with a genetic diagnosis of SMA; long-term safety follow-up of these patients must now be a key priority.