Evidence map›Paper›PMID 40606545›Full record

ArticleMolecular therapy. Methods & clinical development2025

Repeat length as a key determinant for disease severity and antisense oligonucleotide activity in myotonic dystrophy type 1.

Najoua El Boujnouni, Lise Ripken, Marieke Willemse, Bart van der Sanden, Kornelia Neveling, Alexander Hoischen, Roland Brock, Derick G Wansink

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Article
  2. Review
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

8 authors.

Najoua El BoujnouniDepartment of Medical BioSciences, Radboud University Medical Center, Nijmegen 6525 GA, the Netherlands.
Lise RipkenDepartment of Medical BioSciences, Radboud University Medical Center, Nijmegen 6525 GA, the Netherlands.
Marieke WillemseDepartment of Medical BioSciences, Radboud University Medical Center, Nijmegen 6525 GA, the Netherlands.
Bart van der SandenDepartment of Human Genetics, Radboud University Medical Center, Nijmegen 6525 GA, the Netherlands.
Kornelia NevelingDepartment of Human Genetics, Radboud University Medical Center, Nijmegen 6525 GA, the Netherlands.
Alexander HoischenDepartment of Human Genetics, Radboud University Medical Center, Nijmegen 6525 GA, the Netherlands.
Roland BrockDepartment of Medical BioSciences, Radboud University Medical Center, Nijmegen 6525 GA, the Netherlands.
Derick G WansinkDepartment of Medical BioSciences, Radboud University Medical Center, Nijmegen 6525 GA, the Netherlands.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Myotonic dystrophy type 1 (DM1) is caused by a (CTG)

Indexed as

antisense oligonucleotidesCRISPR/Cas9 nickaseDMPKgene editingheterogeneityin vitro modelsmyoblastsmyotonic dystrophy type 1repeat lengthsplicing

Identifiers

PMID40606545
PMCPMC12221585

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.