FDA Approval of Nusinersen for Spinal Muscular Atrophy Makes 2016 the Year of Splice Modulating Oligonucleotides

FDA press release on nusinersen approval. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm534611.htm Accessed on February 3, 2017.

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FDA letter on nusinersen approval to BioGen. www.accessdata.fda.gov/drugsatfda_docs/appletter/2016/209531Orig1s000ltr.pdf Accessed on February 3, 2017.

Briefing document to an SMA Europe, European Medicine Agency and TREAT-NMD stakeholder workshop on SMA therapy development. www.treat-nmd.eu/downloads/file/meetings/2016/SMA_stakeholder_workshop_briefing_document_11.11.2016.pdf Accessed on February 3, 2017.

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Finkel RS , Chiriboga CA , Vajsar J , Day JW , Montes J , De Vivo DC , Yamashita M , Rigo F , Hung G , et al. (2017). Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Lancet, 388:3017–3026.

Press release type I SMA placebo-controlled trial. http://ir.ionispharma.com/phoenix.zhtml?c=222170&p=irol-newsArticle&ID=2191319 Accessed on February 3, 2017.

Press release type II/III SMA placebo-controlled trial. http://ir.ionispharma.com/phoenix.zhtml?c=222170&p=irol-newsArticle&ID=2220037 Accessed on February 3, 2017.

Press release FDA and EMA regulatory status of nusinersen. http://ir.ionispharma.com/phoenix.zhtml?c=222170&p=irol-newsArticle&ID=2217083 Accessed on February 3, 2017.

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Label nusinersen. www.accessdata.fda.gov/drugsatfda_docs/label/2016/209531lbl.pdf Accessed on February 3, 2017.

Label eteplirsen. www.accessdata.fda.gov/drugsatfda_docs/label/2016/206488lbl.pdf Accessed on February 3, 2017.

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