STR1VE (onasemnogene abeparvovec / Zolgensma)
Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion
Single-dose IV AAV9-SMN1 gene therapy produces durable motor benefit in SMA type 1 infants.
Source publication
Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion
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Sponsor
Novartis Gene Therapies
Principal investigator
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Population
SMA - Spinal Muscular Atrophy, Gene Therapy; n=22
Primary endpoint
Achievement of Independent Sitting for at Least 30 Seconds
Methodology notes
Full methodology is under curation. This project aggregates 3 seeded claims across three temporal layers. The replication spec, pre-registered analysis, and version history will appear here as the project matures.
Funding progress
$1.2M / $4.0M
31%
Funding displayed here is indicative of collaborator interest. Sciync does not broker money, grants, or securities.
Temporal layers
Published findings under validation
1
Active replication in progress
1
Forecast horizons pending resolution
1