Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy-Reference-Cited by-同舟云学术

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

Published:2016-02-10 Issue:10 Volume:86 Page:890-897
ISSN:0028-3878
Container-title:Neurology
language:en
Short-container-title:Neurology

Author:

Chiriboga Claudia A.,Swoboda Kathryn J.,Darras Basil T.,Iannaccone Susan T.,Montes Jacqueline,De Vivo Darryl C.,Norris Daniel A.,Bennett C. Frank,Bishop Kathie M.

Publisher

Ovid Technologies (Wolters Kluwer Health)

Subject

Neurology (clinical)

Reference25 articles.

1. Spinal muscular atrophy

2. Spinal Muscular Atrophy: Clinical Classification and Disease Heterogeneity

3. Disruption of an SF2/ASF-dependent exonic splicing enhancer in SMN2 causes spinal muscular atrophy in the absence of SMN1

4. Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model

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