Nusinersen potentially effective in SMA
نویسندگان
چکیده
منابع مشابه
Nusinersen for spinal muscular atrophy
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متن کاملResults from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy.
OBJECTIVE To examine safety, tolerability, pharmacokinetics, and preliminary clinical efficacy of intrathecal nusinersen (previously ISIS-SMNRx), an antisense oligonucleotide designed to alter splicing of SMN2 mRNA, in patients with childhood spinal muscular atrophy (SMA). METHODS Nusinersen was delivered by intrathecal injection to medically stable patients with type 2 and type 3 SMA aged 2-...
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ژورنال
عنوان ژورنال: Nature Reviews Neurology
سال: 2016
ISSN: 1759-4758,1759-4766
DOI: 10.1038/nrneurol.2016.199