Brogidirsen and Exon 44 Skipping for Duchenne Muscular Dystrophy: Advances and Challenges in RNA-Based Therapy.

Abstract readReview

In one paragraph

Review in Genes, 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.

FDA-approved antisense oligonucleotide therapies for duchenne muscular dystrophy: current status and future outlook.RNA biology · 2026
Review
From N-of-1 to versatility in propionic acidemia: Antisense oligonucleotide-mediated skipping of a constitutiveMolecular therapy. Nucleic acids · 2026
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

2 authors.

Annie TangDepartment of Medical Genetics, University of Alberta, 116 St. & 85 Ave., Edmonton, AB T6G 2R3, Canada.

Toshifumi YokotaDepartment of Medical Genetics, University of Alberta, 116 St. & 85 Ave., Edmonton, AB T6G 2R3, Canada.ORCID 0000-0001-6672-6742

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Duchenne muscular dystrophy (DMD) is a severe inherited muscle-wasting disorder that is associated with severe morbidity and mortality globally. Current treatment options have improved the quality of life of patients, but these treatments are only palliative. There is a need for more DMD treatment options. Antisense oligonucleotide (ASO) therapies have emerged as a promising personalized treatment option for patient groups that possess specific mutations. A subset of these therapies can skip over frame-disrupting exons in the

Indexed as

DystrophinExonsGenetic TherapyMuscular Dystrophy, DuchenneOligonucleotidesOligonucleotides, AntisenseAnimalsHumansMorpholinosDMD protein, humanDystrophingolodirsenMorpholinosOligonucleotidesOligonucleotides, Antisenseantisense oligonucleotideantisense therapeuticsbrogidirsenclinical trialduchenne muscular dystrophydystrophinexon 44exon skippingNS-089/NCNP-02splice modulation

Identifiers

PMID40725431

DOI10.3390/genes16070777

PMCPMC12295302

What OpenQuestion 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 OpenQuestion graph.