DOI: 10.1055/s-00000071

Seminars in Neurology

References

Chiriboga CA, Swoboda KJ, Darras BT. , et al.
Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy.

Neurology 2016;
86 (10) 890-897

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