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Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial
- Source :
- Nature Medicine; July 2022, Vol. 28 Issue: 7 p1381-1389, 9p
- Publication Year :
- 2022
-
Abstract
- For presymptomatic infants at risk for SMA type 1, onasemnogene abeparvovec improves motor outcomes, ventilator-free survival, and nutritional/respiratory independence compared with untreated or treated symptomatic patients
Details
- Language :
- English
- ISSN :
- 10788956 and 1546170X
- Volume :
- 28
- Issue :
- 7
- Database :
- Supplemental Index
- Journal :
- Nature Medicine
- Publication Type :
- Periodical
- Accession number :
- ejs60219415
- Full Text :
- https://doi.org/10.1038/s41591-022-01866-4