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

Primary endpoint

Achievement of Independent Sitting for at Least 30 Seconds

Population

SMA - Spinal Muscular Atrophy, Gene Therapy; n=22

Historical

Current

Future

3 noemes

Historical

1 noeme

Published findings from this trial. Vote whether the expert body still believes each noeme stands given current evidence.

The STR1VE (onasemnogene abeparvovec / Zolgensma) trial's stated primary conclusion — Single-dose IV AAV9-SMN1 gene therapy produces durable motor benefit in SMA type 1 infants. — replicates in independent cohorts.

gene-therapy

Current

1 noeme

Noemes tracking active replication, subgroup analysis, or long-term follow-up. Vote on the likelihood the original effect holds.

Recent follow-up analyses of STR1VE (onasemnogene abeparvovec / Zolgensma) are confirming the original effect size in real-world data.

gene-therapy

Future

1 noeme

Open forecast horizons tied to this trial. Vote your probability that each resolves TRUE by the declared horizon.

By 2028, long-term follow-up will show durability of Zolgensma motor benefit plateau at ≥5 years post-infusion in early-treated cohorts.

gene-therapy

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