Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
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Discussion
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Implication
Randomized trial demonstrates improved survival and motor function with nusinersen in infants with spinal muscular atrophy, indicating the importance of early therapeutic intervention.
Key Points
- To assess the clinical efficacy of nusinersen on survival and motor development compared with a sham control in infants with infantile-onset spinal muscular atrophy.
- Randomized, sham-controlled clinical trial (ENDEAR; ClinicalTrials.gov number, NCT02193074).
- Evaluated infants diagnosed with spinal muscular atrophy assigned to nusinersen or sham-control procedures.
- Infants receiving nusinersen demonstrated higher survival rates compared to those in the control group.
- Patients treated with nusinersen achieved superior improvements in motor function relative to sham control, with evidence indicating early treatment optimizes therapeutic benefit.
Cite This Study
Finkel et al. (2017) studied this question.