Forecast horizon — calibration-scored at resolution.
By 2028, universal newborn SMA screening + early nusinersen (or onasemnogene/risdiplam) will be standard across all US states and most EU countries.
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Evidence stream
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Registry data
Apr 18, 2026
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Source publication
An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
Semantically related
Nearest claims in the expert-corpus vector space. Ordered by cosine distance — lower is closer.
0.1835
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.
0.2050
By 2028, long-term follow-up will show durability of Zolgensma motor benefit plateau at ≥5 years post-infusion in early-treated cohorts.
0.2164
By 2028, at least two additional subretinal gene therapies (beyond LUXTURNA) will be FDA-approved for monogenic retinal dystrophies.
0.2220
Recent follow-up analyses of NURTURE (nusinersen) are confirming the original effect size in real-world data.
0.2248
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.
0.2249
By 2028, exa-cel or a comparable ex vivo gene-edited therapy will have treated >500 SCD patients globally with a durable VOC-free rate >90% at 3 years.