NURTURE (nusinersen)
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
Presymptomatic nusinersen in SMA infants preserves motor function milestones in long-term follow-up.
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
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Sponsor
Biogen
Principal investigator
Medical Director
Population
Spinal Muscular Atrophy; n=25
Primary endpoint
Time to Death or Respiratory Intervention
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
$1.4M / $2.7M
51%
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