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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Journal

Journal ID (iso-abbrev): Lancet

Title: Lancet (London, England)

Publisher: Elsevier BV

ISSN (Electronic): 1474-547X

ISSN (Print): 0140-6736

Publication date (Electronic): December 17 2016

Volume: 388

Issue: 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

Publisher Item ID: S0140-6736(16)31408-8

DOI: 10.1016/S0140-6736(16)31408-8

PubMed ID: 27939059

SO-VID: 304efc1c-f0c7-47a3-a608-0a4209fb19e9

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