Evidence map›Paper›PMID 40150900›Full record

ReviewJournal of neuromuscular diseases2026

Antisense RNA therapies for muscular dystrophies.

Virginia Arechavala-Gomeza, Andrea López-Martínez, Annemieke Aartsma-Rus

Abstract readReview
In one paragraph

Review in Journal of neuromuscular diseases, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing 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

7 citing papers in PubMed.

  1. Article
  2. Review
  3. Review
  4. Review
  5. Article
  6. Review
  7. Characterization ofNAR molecular medicine · 2025
    Article
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.

Virginia Arechavala-GomezaNucleic Acid Therapeutics for Rare Diseases (NAT-RD), Biobizkaia Health Research Institute, Barakaldo, Spain.ORCID 0000-0001-7703-3255
Andrea López-MartínezNucleic Acid Therapeutics for Rare Diseases (NAT-RD), Biobizkaia Health Research Institute, Barakaldo, Spain.ORCID 0000-0003-4711-7495
Annemieke Aartsma-RusDepartment of Human Genetics, Leiden University Medical Center, Leiden, the Netherlands.ORCID 0000-0003-1565-654X

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Inherited muscular dystrophies are a heterogeneous group of diseases, caused by different types of genetic mutations. RNA therapies, and particularly antisense oligonucleotides, offer a palette of therapeutic strategies to either reduce the production of harmful proteins or to restore or increase protein expression. Consequently, they offer therapeutic promise for multiple forms of muscular dystrophies. This review outlines the different RNA therapy types considered for the treatment of Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy and myotonic dystrophy, emphasizing the strategies used to deliver these therapies to skeletal muscle with a focus on approaches that have reached the clinical trial stage.

Indexed as

Genetic TherapyMuscular DystrophiesRNA, AntisenseAnimalsHumansMuscular Dystrophy, DuchenneMuscular Dystrophy, FacioscapulohumeralMyotonic DystrophyOligonucleotides, AntisenseOligonucleotides, AntisenseRNA, Antisenseantisense oligonucleotidemuscle deliverymuscular dystrophysiRNA

Identifiers

PMID40150900
PMCPMC13141843

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.