Article
Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study.
Lancet (London, England) - 17 Dec 2016
Finkel Richard S, Chiriboga Claudia A, Vajsar Jiri, Day John W, Montes Jacqueline, De Vivo Darryl C, Yamashita Mason, Rigo Frank, Hung Gene, Schneider Eugene, Norris Daniel A, Xia Shuting, Bennett C Frank, Bishop Kathie M
Abstract excerpt
BACKGROUND: Nusinersen is a 2'-O-methoxyethyl phosphorothioate-modified antisense drug being developed to treat spinal muscular atrophy. Nusinersen is specifically designed to alter splicing of SMN2 pre-mRNA and thus increase the amount of functional survival motor neuron (SMN) protein that is deficient in patients with spinal muscular atrophy. METHODS: This open-label, phase 2, escalating dose clinical study...
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