Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

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...

Podrobná bibliografie
Hlavní autoři:	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
Médium:	Journal article
Jazyk:	English
Vydáno:	Massachusetts Medical Society 2017

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