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July 28, 2020; 95 (4) Editorial

Nusinersen in SMA 2 and 3

Risks vs benefits

Feza Deymeer
First published July 14, 2020, DOI: https://doi.org/10.1212/WNL.0000000000009919
Feza Deymeer
Department of Neurology, Istanbul Medical Faculty, Istanbul University.
MD, MS (Epid)
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Nusinersen in SMA 2 and 3
Risks vs benefits
Feza Deymeer
Neurology Jul 2020, 95 (4) 151-152; DOI: 10.1212/WNL.0000000000009919

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This article has a correction. Please see:

  • Nusinersen in SMA 2 and 3 - November 24, 2020
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Nusinersen is an antisense oligonucleotide which promotes the expression of full-length SMN protein by modifying the splicing of SMN2 pre-messenger RNA. Nusinersen was approved by FDA in 2016 to be used in all forms of SMA, based on a clinical trial in SMA 1.1 Two years later, a clinical trial in later-onset SMA was published.2 Ability to walk independently ≥15 feet was a criterion of exclusion in this trial so that the study was mainly in children with SMA 2. No clinical trial was published in SMA 3.

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  • Go to Neurology.org/N for full disclosures. Funding information and disclosures deemed relevant by the authors, if any, are provided at the end of the article.

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  • © 2020 American Academy of Neurology
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