Randomized trial demonstrates improved survival and motor function with nusinersen in infants with spinal muscular atrophy, indicating the importance of early therapeutic intervention.
Among infants with spinal muscular atrophy, those who received nusinersen were more likely to be alive and have improvements in motor function than those in the control group. Early treatment may be necessary to maximize the benefit of the drug. (Funded by Biogen and Ionis Pharmaceuticals; ENDEAR ClinicalTrials.gov number, NCT02193074 .).
No takes yet. Share an insight, caveat, or question.
Finkel et al. (2017) studied this question.
Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context: