February 11, 2016Open Access

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

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Abstract

Objective: To examine safety, tolerability, pharmacokinetics, and preliminary clinical efficacy of intrathecal nusinersen (previously ISIS-SMN Rx ), an antisense oligonucleotide designed to alter splicing of SMN2 mRNA, in patients with childhood spinal muscular atrophy (SMA).

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Authors

Claudia A. Chiriboga

Università Cattolica del Sacro Cuore

Kathryn J. Swoboda

Massachusetts General Hospital

Basil T. Darras

Università Cattolica del Sacro Cuore

Journals

Neurology

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Institutions

The University of Texas Southwestern Medical Center

University of Utah

Boston Children's Hospital

References and Citations

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

Key Points

Abstract

Citation Network

Connected Papers

Discussion

Authors

Journals

Actions

Institutions

References and Citations

Citation Network

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Discussion

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