Evidence map›Paper›PMID 37751291›Full record

ArticleJCI insight2023

Dimethyl fumarate modulates the dystrophic disease program following short-term treatment.

Cara A Timpani, Stephanie Kourakis, Danielle A Debruin, Dean G Campelj, Nancy Pompeani, Narges Dargahi, Angelo P Bautista, Ryan M Bagaric, Elya J Ritenis, Lauren Sahakian and 10 more

Open access · goldAbstract read
In one paragraph

Article in JCI insight, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed
1.8field-weighted citation impact, top 14% of its field
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

9 citing papers in PubMed, 12 citations in OpenAlex.

  1. Article
  2. Article
  3. Article
  4. Review
  5. Review
  6. Article
  7. Review
  8. MCU-independent CaScientific reports · 2024
    Article
  9. Adenylosuccinic Acid: An Orphan Drug with Untapped Potential.Pharmaceuticals (Basel, Switzerland) · 2023
    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

20 authors at 9 institutions in 2 countries.

Cara A TimpaniInstitute for Health and Sport (IHeS), Victoria University, Melbourne, Victoria, Australia.
Stephanie KourakisInstitute for Health and Sport (IHeS), Victoria University, Melbourne, Victoria, Australia.
Danielle A DebruinInstitute for Health and Sport (IHeS), Victoria University, Melbourne, Victoria, Australia.
Dean G CampeljInstitute for Health and Sport (IHeS), Victoria University, Melbourne, Victoria, Australia.
Nancy PompeaniInstitute for Health and Sport (IHeS), Victoria University, Melbourne, Victoria, Australia.
Narges DargahiInstitute for Health and Sport (IHeS), Victoria University, Melbourne, Victoria, Australia.
Angelo P BautistaSchool of Molecular Sciences, The University of Western Australia, Perth, Western Australia, Australia.
Ryan M BagaricInstitute for Health and Sport (IHeS), Victoria University, Melbourne, Victoria, Australia.
Elya J RitenisCollege of Health and Biomedicine, Victoria University, Melbourne, Victoria, Australia.
Lauren SahakianInstitute for Health and Sport (IHeS), Victoria University, Melbourne, Victoria, Australia.
Didier DebrincatCollege of Health and Biomedicine, Victoria University, Melbourne, Victoria, Australia.
Nicole StupkaInstitute for Health and Sport (IHeS), Victoria University, Melbourne, Victoria, Australia.
Patricia HafnerDivision of Neuropaediatrics and Developmental Medicine, University Children's Hospital of Basel (UKBB), Basel, Switzerland.
Peter G ArthurSchool of Molecular Sciences, The University of Western Australia, Perth, Western Australia, Australia.
Jessica R TerrillSchool of Molecular Sciences, The University of Western Australia, Perth, Western Australia, Australia.
Vasso ApostolopoulosInstitute for Health and Sport (IHeS), Victoria University, Melbourne, Victoria, Australia.
Judy B de HaanBasic Science Domain, Oxidative Stress Laboratory, Baker Heart and Diabetes Institute, Melbourne, Victoria, Australia.
Nuri GuvenDepartment of Medicine - Western Health, Melbourne Medical School, The University of Melbourne, St Albans, Victoria, Australia.
Dirk FischerDivision of Neuropaediatrics and Developmental Medicine, University Children's Hospital of Basel (UKBB), Basel, Switzerland.
Emma RybalkaInstitute for Health and Sport (IHeS), Victoria University, Melbourne, Victoria, Australia.
Victoria University · AUAustralian Institute for Musculoskeletal Science · AUThe University of Western Australia · AUWestern Health · AUThe University of Melbourne · AUUniversity of Basel · CHBaker Heart and Diabetes Institute · AUFlorey Institute of Neuroscience and Mental Health · AUUniversity of Tasmania · AU

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

New medicines are urgently required to treat the fatal neuromuscular disease Duchenne muscular dystrophy (DMD). Dimethyl fumarate (DMF) is a potent immunomodulatory small molecule nuclear erythroid 2-related factor 2 activator with current clinical utility in the treatment of multiple sclerosis and psoriasis that could be effective for DMD and rapidly translatable. Here, we tested 2 weeks of daily 100 mg/kg DMF versus 5 mg/kg standard-care prednisone (PRED) treatment in juvenile mdx mice with early symptomatic DMD. Both drugs modulated seed genes driving the DMD disease program and improved force production in fast-twitch muscle. However, only DMF showed pro-mitochondrial effects, protected contracting muscles from fatigue, improved histopathology, and augmented clinically compatible muscle function tests. DMF may be a more selective modulator of the DMD disease program than PRED, warranting follow-up longitudinal studies to evaluate disease-modifying impact.

Indexed as

Dimethyl FumarateMuscular Dystrophy, DuchenneAnimalsMiceMice, Inbred mdxMusclesPrednisoneDimethyl FumaratePrednisoneDrug therapyMuscle BiologyNeuromuscular diseaseSkeletal muscleTherapeutics

Identifiers

PMID37751291
PMCPMC10721277
OpenAlexW4387033212

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.