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Nusinersen in SMA 2 and 3

Name: Nusinersen in SMA 2 and 3
Creator: Feza Deymeer
Keywords: Muscular Atrophy, Spinal, 03 medical and health sciences, 0302 clinical medicine, Oligonucleotides, Humans, 3. Good health

Risks vs benefits

descriptionPublicationkeyboard_double_arrow_right Article , Other literature type 28 Jul 2020 English Publisher:Ovid Technologies (Wolters Kluwer Health)Journal:Neurology, volume 95, pages 151-152 (issn: 0028-3878, eissn: 1526-632X,

Authors: Feza Deymeer;

doi: 10.1212/wnl.0000000000009919

pmid: 32665409

Nusinersen in SMA 2 and 3

- Summary
- Subjects
- Metrics

Abstract

Nusinersen is an antisense oligonucleotide which promotes the expression of full-length SMN protein by modifying the splicing of SMN2 pre-messenger RNA. Nusinersen was approved by FDA in 2016 to be used in all forms of SMA, based on a clinical trial in SMA 1.1 Two years later, a clinical trial in later-onset SMA was published.2 Ability to walk independently ≥15 feet was a criterion of exclusion in this trial so that the study was mainly in children with SMA 2. No clinical trial was published in SMA 3.

Related Organizations

Istanbul University
Turkey

Keywords

Muscular Atrophy, Spinal, Oligonucleotides, Humans

Impact byBIP!

	citations This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).	2
	popularity This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.	Average
	influence This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).	Top 10%
	impulse This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.	Average