Published finding — does the expert body still believe it?
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.
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Evidence stream
1 event · 0 snapshots
Registry data
Apr 18, 2026
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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
Semantically related
Nearest claims in the expert-corpus vector space. Ordered by cosine distance — lower is closer.
0.1052
Recent follow-up analyses of STR1VE (onasemnogene abeparvovec / Zolgensma) are confirming the original effect size in real-world data.
0.1178
The NURTURE (nusinersen) trial's stated primary conclusion — Presymptomatic nusinersen in SMA infants preserves motor function milestones in long-term follow-up. — replicates in independent cohorts.
0.1904
Recent follow-up analyses of NURTURE (nusinersen) are confirming the original effect size in real-world data.
0.2037
The LUXTURNA (voretigene) trial's stated primary conclusion — Subretinal AAV2-hRPE65 gene therapy improves functional vision in biallelic RPE65-associated inherited retinal dystrophy. — replicates in independent cohorts.
0.2089
The HOPE-B (Hemgenix / etranacogene) trial's stated primary conclusion — AAV5 FIX-Padua gene therapy produces sustained Factor IX activity, reducing bleeds in severe hemophilia B. — replicates in independent cohorts.
0.2143
The HGB-206 (lovo-cel) trial's stated primary conclusion — Lentiviral β-globin gene therapy reduces or eliminates VOC events in severe sickle cell disease. — replicates in independent cohorts.