Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
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Author / Creator
ENDEAR Study Group , Finkel, Richard S , Mercuri, Eugenio , Darras, Basil T , Connolly, Anne M , Kuntz, Nancy L , Kirschner, Janbernd , Chiriboga, Claudia A , Saito, Kayoko , Servais, Laurent , Tizzano, Eduardo , Topaloglu, Haluk , Tulinius, Már , Montes, Jacqueline , Glanzman, Allan M , Bishop, Kathie , Zhong, Z. John , Gheuens, Sarah , Bennett, C. Frank , Schneider, Eugene , Farwell, Wildon and De Vivo, Darryl C
Publisher
United States: Massachusetts Medical Society
Journal title
Language
English
Formats
Publication information
Publisher
United States: Massachusetts Medical Society
Subjects
More information
Scope and Contents
Contents
In this phase 3 trial, among infants with spinal muscular atrophy, those who received nusinersen were more likely to achieve major motor milestones and less likely to need permanent assisted ventilation than those who underwent a sham procedure.
Alternative Titles
Full title
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
Authors, Artists and Contributors
Author / Creator
Finkel, Richard S
Mercuri, Eugenio
Darras, Basil T
Connolly, Anne M
Kuntz, Nancy L
Kirschner, Janbernd
Chiriboga, Claudia A
Saito, Kayoko
Servais, Laurent
Tizzano, Eduardo
Topaloglu, Haluk
Tulinius, Már
Montes, Jacqueline
Glanzman, Allan M
Bishop, Kathie
Zhong, Z. John
Gheuens, Sarah
Bennett, C. Frank
Schneider, Eugene
Farwell, Wildon
De Vivo, Darryl C
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_swepub_primary_oai_gup_ub_gu_se_259613
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_swepub_primary_oai_gup_ub_gu_se_259613
Other Identifiers
ISSN
0028-4793,1533-4406
E-ISSN
1533-4406
DOI
10.1056/NEJMoa1702752
