Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial
Clinic for Special Children · University of Massachusetts Chan Medical School · +22 more institutions
Abstract
Most children with biallelic SMN1 deletions and three SMN2 copies develop spinal muscular atrophy (SMA) type 2. SPR1NT ( NCT03505099 ), a Phase III, multicenter, single-arm trial, investigated the efficacy and safety of onasemnogene abeparvovec for presymptomatic children with biallelic SMN1 mutations treated within six postnatal weeks. Of 15 children with three SMN2 copies treated before symptom onset, all stood independently before 24 months (P
Citation impact
- FWCI
- 36.46
- Percentile
- 100%
- References
- 44
Authors
21- KAKevin A. StraussCorresponding
Clinic for Special Children, University of Massachusetts Chan Medical School, Lancaster General Hospital
- MAMichelle A. Farrar
UNSW Sydney, Sydney Children’s Hospitals Network
- FMFrancesco Muntoni
Great Ormond Street Hospital, University College London
- KSKayoko Saito
Tokyo Women's Medical University
- JRJerry R. Mendell
Nationwide Children's Hospital, The Ohio State University
Topics & keywords
- Spinal muscular atrophy
- Medicine
- Atrophy
- Pediatrics
- Internal medicine
- Disease
- Zero hunger