Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

Spinal muscular atrophy is an autosomal recessive neuromuscular disorder that is caused by an insufficient level of survival motor neuron (SMN) protein. Nusinersen is an antisense oligonucleotide drug that modifies pre–messenger RNA splicing of the SMN2 gene and thus promotes increased production of...

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Bibliografiske detaljer
Main Authors: Richard S. Finkel, Eugenio Mercuri, Basil T. Darras, Anne M. Connolly, Nancy L. Kuntz, Janbernd Kirschner, Claudia A. Chiriboga, Kayoko Saito, Laurent Servais, Eduardo F. Tizzano, Haluk Topaloğlu, M. Tulinius, Jacqueline Montes, Allan M. Glanzman, Kathie M. Bishop, Zhensbao Zhong, Sarah Gheuens, C. Frank Bennett, Eugene Schneider, Wildon Farwell, Darryl C. De Vivo
Format: Artigo
Sprog:engelsk
Udgivet: 2017
Online adgang:https://doi.org/10.1056/nejmoa1702752
https://www.nejm.org/doi/pdf/10.1056/NEJMoa1702752?articleTools=true
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https://doi.org/10.1056/nejmoa1702752
https://www.nejm.org/doi/pdf/10.1056/NEJMoa1702752?articleTools=true

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