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NEUROMUSCULAR DISEASE

Long-term benefits of nusinersen in later-onset spinal muscular atrophy

Nature Reviews Neurology volume 15, pages 368–369 (2019)Cite this article

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Treatment with the antisense oligonucleotide nusinersen benefits patients with spinal muscular atrophy (SMA) type II and type III over 3 years, a new study shows. The results not only demonstrate the potential of nusinersen in older children, but also reveal that improvements continue over time in patients receiving this drug.

SMA is a genetic neuromuscular disorder. The most common form is SMA type I, which has an age of onset <6 months, severely impairs motor development and leads to death. SMA types II and III have later ages of onset (7–18 months and >18 months, respectively), and patients can lose existing motor function over time.

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Change history

  • 17 May 2019

    In the original version of this article, the abbreviation CMAP was misspelt as CAMP. The spelling has been corrected in the HTML and PDF versions of the article.

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Further reading

  • Groen, E. J. et al. Advances in therapy for spinal muscular atrophy: promises and challenges. Nat. Rev. Neurol. 14, 214–224 (2018)

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  1. Nature Reviews Neurology http://www.nature.com/nrneurol/

    Ian Fyfe

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  1. Ian Fyfe
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Correspondence to Ian Fyfe.

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Fyfe, I. Long-term benefits of nusinersen in later-onset spinal muscular atrophy. Nat Rev Neurol 15, 368–369 (2019). https://doi.org/10.1038/s41582-019-0202-4

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  • DOI: https://doi.org/10.1038/s41582-019-0202-4

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