Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy.

Volume: 86, Issue: 10, Pages: 890 - 897
Published: Mar 8, 2016
Abstract
Objective: null To examine safety, tolerability, pharmacokinetics, and preliminary clinical efficacy of intrathecal nusinersen (previously ISIS-SMN Rx ), an antisense oligonucleotide designed to alter splicing of null SMN2 null mRNA, in patients with childhood spinal muscular atrophy (SMA). null Methods: null Nusinersen was delivered by intrathecal injection to medically stable patients with type 2 and type 3 SMA aged 2–14 years in an open-label...
Paper Details
Title
Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy.
Published Date
Mar 8, 2016
Journal
Volume
86
Issue
10
Pages
890 - 897
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