Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with #spinalmuscularatrophy #SMA

Originally posted 2016-08-02 10:50:22.

Nusinersen (previously ISIS-SMNRx and also known as ISIS 396443) is an antisense oligonucleotide designed to bind to the SMN2 pre-mRNA and promote inclusion of exon 7 (figure 1).6,7 In mouse models of SMA, nusinersen enhanced exon 7 inclusion, increased SMN protein production, and improved function.6,–9 Nusinersen is delivered by intrathecal injection as antisense oligonucleotide drugs do not cross an intact blood–brain barrier when delivered systemically.10 This first-in-human, open-label, single-ascending dose study was designed to assess safety, tolerability, pharmacokinetics, and clinical effects of intrathecal nusinersen in patients with childhood SMA.

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