Evidence map›Paper›PMID 42568847›Full record

ArticleMolecular therapy. Nucleic acids2026

Timing matters: Exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy.

Sofia Stenler, Junyu Huang, Tirsa L E van Westering, Anna M L Coenen-Stass, Yahya Jad, Kaarel Krjutškov, Samir El Andaloussi, Matthew J A Wood, Thomas C Roberts

Abstract read
In one paragraph

Article in Molecular therapy. Nucleic acids, 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

9 authors.

Sofia StenlerDepartment of Physiology, Anatomy and Genetics, University of Oxford, South Parks Road, Oxford OX1 3QX, UK.
Junyu HuangDepartment of Paediatrics, University of Oxford, South Parks Road, Oxford OX1 3QX, UK.
Tirsa L E van WesteringDepartment of Physiology, Anatomy and Genetics, University of Oxford, South Parks Road, Oxford OX1 3QX, UK.
Anna M L Coenen-StassDepartment of Physiology, Anatomy and Genetics, University of Oxford, South Parks Road, Oxford OX1 3QX, UK.
Yahya JadDepartment of Paediatrics, University of Oxford, South Parks Road, Oxford OX1 3QX, UK.
Kaarel KrjutškovCelvia CC AS, 50411 Tartu, Estonia.
Samir El AndaloussiDepartment of Laboratory Medicine, Karolinska Institutet, 141 86 Huddinge, Sweden.
Matthew J A WoodDepartment of Physiology, Anatomy and Genetics, University of Oxford, South Parks Road, Oxford OX1 3QX, UK.
Thomas C RobertsDepartment of Physiology, Anatomy and Genetics, University of Oxford, South Parks Road, Oxford OX1 3QX, UK.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Exon skipping is a leading therapeutic approach for Duchenne muscular dystrophy (DMD), whereby modulation of pre-mRNA splicing is used to restore the dystrophin translation reading frame. Four exon skipping drugs have received FDA accelerated approval, despite limited clinical efficacy. To investigate how treatment timing influences exon skipping outcomes, dystrophin-deficient

Indexed as

DMDdystrophinexon skippingMT: Oligonucleotides: Therapies and ApplicationsPPMOtreatment timing

Identifiers

PMID42568847
PMCPMC13448210

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Registered trials

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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 OpenQuestion graph.