Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

<br><strong>Background: </strong>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...

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Bibliographic Details
Main Authors:	Finkel, RS, Mercuri, E, Darras, BT, Connolly, AM, Kuntz, NL, Kirschner, J, Chiriboga, CA, Saito, K, Laurent Servais, Tizzano, E, Topaloglu, H, Tulinius, M, Montes, J, Glanzman, AG, Bishop, K, Zhong, ZJ, Gheuens, S, Bennett, CF, Schneider, E, Farwell, W, De Vivo, DC, ENDEAR Study Group
Format:	Journal article
Language:	English
Published:	Massachusetts Medical Society 2017

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

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