DOI:
10.1055/s-00000041
Neuropediatrics
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References
Finkel RS, 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 (10063): 3017-3026
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