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      Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study.

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          Abstract

          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.

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          Author and article information

          Journal
          Lancet
          Lancet (London, England)
          Elsevier BV
          1474-547X
          0140-6736
          December 17 2016
          : 388
          : 10063
          Affiliations
          [1 ] Nemours Children's Hospital, Orlando, FL, USA. Electronic address: rfinkel@nemours.org.
          [2 ] Columbia University Medical Center, New York, NY, USA.
          [3 ] University of Toronto, Hospital for Sick Children, Toronto, ON, Canada.
          [4 ] Stanford University School of Medicine, Stanford, CA, USA.
          [5 ] Ionis Pharmaceuticals, Inc, Carlsbad, CA, USA.
          Article
          S0140-6736(16)31408-8
          10.1016/S0140-6736(16)31408-8
          27939059
          304efc1c-f0c7-47a3-a608-0a4209fb19e9

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