Evidence map›Paper›PMID 40831143›Full record

ArticleJournal of neuromuscular diseases2026

Association between exon-skipping therapy with eteplirsen and cardiac outcomes in Duchenne muscular dystrophy.

Joel Iff, Isabelle Desguerre, Yunjuan Liu, Francois Sarkozy, Edward Tuttle, Francesco Muntoni, Craig M McDonald, Marie-Christine Nougues, Helge Amthor, Yi Zhong and 2 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article 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 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing 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

6 citing papers in PubMed.

  1. Trial
  2. Article
  3. Article
  4. Review
  5. Article
  6. 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

12 authors.

Joel IffSarepta Therapeutics, Inc, Cambridge, MA, USA.
Isabelle DesguerreAPHP- Necker Hospital, Neuromuscular Center, FILNEMUS Network, Paris, France.
Yunjuan LiuAnalysis Group, Inc, Menlo Park, California, USA.
Francois SarkozyFSNB Health & Care, Paris, France.
Edward TuttleAnalysis Group, Inc, Menlo Park, California, USA.
Francesco MuntoniDubowitz Neuromuscular Centre, University College London, Great Ormond Street Institute of Child Health, London, UK.
Craig M McDonaldDepartments of Physical Medicine & Rehabilitation and Pediatrics, University of California Davis Health, Sacramento, California, USA.
Marie-Christine NouguesDépartement de Neuropédiatrie, Centre de Référence des Pathologies Neuromusculaires, Hôpital Trousseau, APHP, Paris, France.
Helge AmthorService de Neurologie et Réanimation Pédiatrique, Hôpital Raymond Poincaré, APHP Université Paris-Saclay, Garches, France.
Yi ZhongAnalysis Group, Inc, Menlo Park, California, USA.
Karim WahbiCardiology Department, AP-HP, Cochin Hospital, Paris Cedex 14, France.
Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study Investigators

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

BackgroundDuchenne muscular dystrophy (DMD) leads to dilated cardiomyopathy and heart failure during teenage years or young adulthood. Eteplirsen promotes dystrophin production through skipping of exon 51 of the

Indexed as

Cardiomyopathy, DilatedHeart FailureMorpholinosMuscular Dystrophy, DuchenneAdolescentChildDystrophinExonsHumansMaleYoung AdultDystrophineteplirsenMorpholinoscardiomyopathyDuchenne muscular dystrophyeteplirsenexon-skippingheart failureleft ventricular ejection fraction

Identifiers

PMID40831143
PMCPMC13141851

What OpenQuestion holds

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LicenceCC BY-NC
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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.