Evidence map›Paper›PMID 37435300›Full record

ReviewFrontiers in physiology2023

Duchenne muscular dystrophy: disease mechanism and therapeutic strategies.

Addeli Bez Batti Angulski, Nora Hosny, Houda Cohen, Ashley A Martin, Dongwoo Hahn, Jack Bauer, Joseph M Metzger

Open access · goldAbstract readReview
In one paragraph

Review in Frontiers in physiology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 105 papers.

0numbers the graph read from it
0cells of the map it votes in
105citing papers in PubMed
21.9field-weighted citation impact, top 1% 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

105 citing papers in PubMed, 141 citations in OpenAlex.

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  19. CTRP1 regulates skeletal muscle differentiation through quality control of mitochondrial dynamics and function.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  20. Observational

45 more citing papers are in PubMed but not listed here.

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

7 authors at 1 institution in 1 country.

Addeli Bez Batti AngulskiDepartment of Integrative Biology and Physiology, University of Minnesota Medical School, Minneapolis, MN, United States.
Nora HosnyDepartment of Integrative Biology and Physiology, University of Minnesota Medical School, Minneapolis, MN, United States.
Houda CohenDepartment of Integrative Biology and Physiology, University of Minnesota Medical School, Minneapolis, MN, United States.
Ashley A MartinDepartment of Integrative Biology and Physiology, University of Minnesota Medical School, Minneapolis, MN, United States.
Dongwoo HahnDepartment of Integrative Biology and Physiology, University of Minnesota Medical School, Minneapolis, MN, United States.
Jack BauerDepartment of Integrative Biology and Physiology, University of Minnesota Medical School, Minneapolis, MN, United States.
Joseph M MetzgerDepartment of Integrative Biology and Physiology, University of Minnesota Medical School, Minneapolis, MN, United States.
University of Minnesota Medical Center · US

Funding

Myofilaments as regulators of heart function in diseaseR01HL132874 · NHLBI · UNIVERSITY OF MINNESOTA · PI METZGER, JOSEPH MARK · 2017 to 2025
$3.7M
Skeletal muscle sarcomere function in health and diseaseR01AR079477 · NIAMS · UNIVERSITY OF MINNESOTA · PI JOSEPH Mark METZGER · 2022 to 2026
$2.6M
NHLBI NIH HHS R01 HL132874NIAMS NIH HHS R01 AR079477
6 · The paper itself

Abstract

Duchenne muscular dystrophy (DMD) is a severe, progressive, and ultimately fatal disease of skeletal muscle wasting, respiratory insufficiency, and cardiomyopathy. The identification of the dystrophin gene as central to DMD pathogenesis has led to the understanding of the muscle membrane and the proteins involved in membrane stability as the focal point of the disease. The lessons learned from decades of research in human genetics, biochemistry, and physiology have culminated in establishing the myriad functionalities of dystrophin in striated muscle biology. Here, we review the pathophysiological basis of DMD and discuss recent progress toward the development of therapeutic strategies for DMD that are currently close to or are in human clinical trials. The first section of the review focuses on DMD and the mechanisms contributing to membrane instability, inflammation, and fibrosis. The second section discusses therapeutic strategies currently used to treat DMD. This includes a focus on outlining the strengths and limitations of approaches directed at correcting the genetic defect through dystrophin gene replacement, modification, repair, and/or a range of dystrophin-independent approaches. The final section highlights the different therapeutic strategies for DMD currently in clinical trials.

Indexed as

Duchenne muscular dystrophydystrophinmuscle diseasepathophysiologyskeletal muscletherapeutic strategies

Identifiers

PMID37435300
PMCPMC10330733
OpenAlexW4382069925

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.