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- W3043504013 abstract "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." @default.
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- W3043504013 date "2020-07-14" @default.
- W3043504013 modified "2023-09-24" @default.
- W3043504013 title "Nusinersen in SMA 2 and 3" @default.
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- W3043504013 doi "https://doi.org/10.1212/wnl.0000000000009919" @default.
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