DOI: 10.1055/s-00000140

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References

Finkel R, Chiriboga CA, Vajsar J. et al.
Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study.

Lancet 2016;
388: 3017-3026
doi:10.1016/S0140-6736(16)31408-8

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