Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial
Clinic for Special Children · University of Massachusetts Chan Medical School · +22 more institutions
Abstract
SPR1NT ( NCT03505099 ) was a Phase III, multicenter, single-arm study to investigate the efficacy and safety of onasemnogene abeparvovec for presymptomatic children with biallelic SMN1 mutations treated at ≤6 weeks of life. Here, we report final results for 14 children with two copies of SMN2, expected to develop spinal muscular atrophy (SMA) type 1. Efficacy was compared with a matched Pediatric Neuromuscular Clinical Research natural-history cohort (n = 23). All 14 enrolled infants sat independently for ≥30 seconds at any visit ≤18 months (Bayley-III item #26; P
Citation impact
- FWCI
- 39.35
- Percentile
- 100%
- References
- 57
Authors
20- 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
- SMN1
- Medicine
- SMA*
- Pediatrics
- Cohort
- Clinical trial
- Adverse effect
- Zero hunger
Funding
- CMChildren's Medical Research
- BBiogen
- APAlexion Pharmaceuticals
- CHChildren's Hospital of Philadelphia
- PTPTC Therapeutics
- CCytokinetics
- AAveXis
- STSarepta Therapeutics
- UOUniversity of New South Wales
- UOUniversity of Sydney
- GGrifols
- NSNew South Wales Government
- GGenentech
- CPChugai Pharmaceutical
- NINovartis Institutes for BioMedical Research
- UPUCB Pharma