Early treatment is a lifeline for infants with SMA.
editorial · Level V
Where this comes from
- Record sourced from PubMed, PMID 35840728.
- Also identified by DOI 10.1038/s41591-022-01889-x and PMC identifier 10228631.
- No licence information is recorded for this record.
- Because redistribution is not established, this page shows the abstract only. Follow the links below for the full text.
Abstract
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.
Medical subject headings
- Muscular Atrophy, Spinal