Nusinersen versus sham control in later-onset spinal muscular atrophy

Nusinersen versus sham control in later-onset spinal muscular atrophy

<p><strong>Background:</strong><br /> Nusinersen is an antisense oligonucleotide drug that modulates pre–messenger RNA splicing of the survival motor neuron 2 (SMN2) gene. It has been developed for the treatment of spinal muscular atrophy (SMA).</p><br /> <p&g...

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Bibliographische Detailangaben
Hauptverfasser: Mercuri, E, Darras, BT, Chiriboga, CA, Day, JW, Campbell, C, Connolly, AM, Iannaccone, ST, Kirschner, J, Kuntz, NL, Saito, K, Shieh, PB, Tulinius, M, Mazzone, ES, Montes, J, Bishop, KM, Yang, Q, Foster, R, Gheuens, S, Bennett, CF, Farwell, W, Schneider, E, De Vivo, DC, Finkel, RS
Format: Journal article
Sprache:English
Veröffentlicht: Massachusetts Medical Society 2018

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