Evidence map›Paper›PMID 42149739›Full record

ReviewRNA biology2026

Nusinersen: the antisense oligonucleotide at the forefront of spinal muscular atrophy treatment.

Natalia N Singh, Eric W Ottesen, Ravindra N Singh

Abstract readReview
In one paragraph

Review in RNA biology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

3 authors.

Natalia N SinghDepartment of Biomedical Sciences, Iowa State University, Ames, IA, USA.ORCID 0000-0002-4846-6892
Eric W OttesenDepartment of Biomedical Sciences, Iowa State University, Ames, IA, USA.ORCID 0000-0003-3303-6280
Ravindra N SinghDepartment of Biomedical Sciences, Iowa State University, Ames, IA, USA.ORCID 0000-0001-5399-2662

Funding

Splicing Regulation of Spinal Muscular Atrophy GenesR01NS055925 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI SINGH, RAVINDRA N · 2006 to 2023
$5.6M
Transcriptome-Wide Effect of Therapeutic Antisense Oligonucleotides for the Treatment of Spinal Muscular AtrophyR03NS136717 · NINDS · IOWA STATE UNIVERSITY · PI SINGH, RAVINDRA N · 2024 to 2025
$153k
NINDS NIH HHS R01 NS055925NINDS NIH HHS R03 NS136717
6 · The paper itself

Abstract

Spinal muscular atrophy (SMA) is the first human disease to be treated with an antisense oligonucleotide (ASO) that restores the full coding sequence of a mRNA through splicing modulation. The therapeutic ASO nusinersen (marketed as Spinraza

Indexed as

Muscular Atrophy, SpinalOligonucleotidesOligonucleotides, AntisenseAnimalsExonsHumansRNA SplicingSurvival of Motor Neuron 2 ProteinnusinersenOligonucleotidesOligonucleotides, AntisenseSMN2 protein, humanSurvival of Motor Neuron 2 Protein2‘-O-methoxyethyl modificationantisense oligonucleotideNusinersenSMA; survival motor neuronSMN; ISS-N1Spinal muscular atrophySpinrazaTM

Identifiers

PMID42149739
PMCPMC13203021

What Socratic holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.