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

PI Medical Director

Primary endpoint

Time to Death or Respiratory Intervention

Population

Spinal Muscular Atrophy; n=25

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 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.

neurology

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 NURTURE (nusinersen) are confirming the original effect size in real-world data.

neurology

Future

1 noeme

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

By 2028, universal newborn SMA screening + early nusinersen (or onasemnogene/risdiplam) will be standard across all US states and most EU countries.

neurology

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