Nusinersen in SMA 2 and 3
Risks vs benefits
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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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