Evidence map›Paper›PMID 28974147›Full record

ReviewToxicologic pathology2017

Duchenne and Becker Muscular Dystrophies: A Review of Animal Models, Clinical End Points, and Biomarker Quantification.

Kristin Wilson, Crystal Faelan, Janet C Patterson-Kane, Daniel G Rudmann, Steven A Moore, Diane Frank, Jay Charleston, Jon Tinsley, G David Young, Anthony J Milici

Open access · bronzeAbstract readReview
In one paragraph

Review in Toxicologic pathology, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 40 papers.

0numbers the graph read from it
0cells of the map it votes in
40citing papers in PubMed
2.5field-weighted citation impact, top 10% 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

40 citing papers in PubMed, 66 citations in OpenAlex.

  1. Trial
  2. 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
  3. Article
  4. Local Non-Coding Regulatory Elements in Muscular Dystrophies.International journal of molecular sciences · 2025
    Review
  5. Article
  6. Article
  7. Article
  8. Article
  9. Article
  10. Article
  11. Caveolin and NOS in the Development of Muscular Dystrophy.International journal of molecular sciences · 2024
    Review
  12. Review
  13. Review
  14. Article
  15. Article
  16. Review
  17. [Pathologic features of paraspinal muscle biopsies in patients with adolescent idiopathic scoliosis].Beijing da xue xue bao. Yi xue ban = Journal of Peking University. Health sciences · 2023
    Article
  18. Article
  19. Review
  20. Dystrophin (International journal of molecular sciences · 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

10 authors at 3 institutions in 2 countries.

Kristin Wilson1 Flagship Biosciences, Inc., Westminster, Colorado, USA.
Crystal Faelan1 Flagship Biosciences, Inc., Westminster, Colorado, USA.
Janet C Patterson-Kane1 Flagship Biosciences, Inc., Westminster, Colorado, USA.
Daniel G Rudmann1 Flagship Biosciences, Inc., Westminster, Colorado, USA.
Steven A Moore2 Department of Pathology, Carver College of Medicine, The University of Iowa, Iowa City, Iowa, USA.
Diane Frank3 Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA.
Jay Charleston3 Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA.
Jon Tinsley4 Summit Therapeutics, Abingdon, United Kingdom.
G David Young1 Flagship Biosciences, Inc., Westminster, Colorado, USA.
Anthony J Milici1 Flagship Biosciences, Inc., Westminster, Colorado, USA.
Sarepta Therapeutics (United States) · USSummit (United Kingdom) · GBUniversity of Iowa · US

Funding

Therapeutic Potential of ESC-Derived Myogenic PrecursorsU54NS053672 · NINDS · UNIVERSITY OF IOWA · PI CAMPBELL, KEVIN P. · 2005 to 2019
$22.0M
NINDS NIH HHS U54 NS053672
6 · The paper itself

Abstract

Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) are neuromuscular disorders that primarily affect boys due to an X-linked mutation in the DMD gene, resulting in reduced to near absence of dystrophin or expression of truncated forms of dystrophin. Some newer therapeutic interventions aim to increase sarcolemmal dystrophin expression, and accurate dystrophin quantification is critical for demonstrating pharmacodynamic relationships in preclinical studies and clinical trials. Current challenges with measuring dystrophin include the variation in protein expression within individual muscle fibers and across whole muscle samples, the presence of preexisting dystrophin-positive revertant fibers, and trace amounts of residual dystrophin. Immunofluorescence quantification of dystrophin can overcome many of these challenges, but manual quantification of protein expression may be complicated by variations in the collection of images, reproducible scoring of fluorescent intensity, and bias introduced by manual scoring of typically only a few high-power fields. This review highlights the pathology of DMD and BMD, discusses animal models of DMD and BMD, and describes dystrophin biomarker quantitation in DMD and BMD, with several image analysis approaches, including a new automated method that evaluates protein expression of individual muscle fibers.

Indexed as

Endpoint DeterminationAnimalsBiomarkersDisease Models, AnimalDystrophinGene Expression RegulationHumansMuscle Fibers, SkeletalMuscular Dystrophy, DuchenneMutationUtrophinBiomarkersDystrophinUtrophinanimal modelscomputational tissue analysisDuchenne muscular dystrophydystrophinimage analysislamininutrophin

Identifiers

PMID28974147
PMCPMC5788182
OpenAlexW2763941029

What OpenQuestion holds

Textmetadata
LicenceTDM
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.