Evidence map›Paper›PMID 33602943›Full record

ReviewNature reviews. Disease primers2021

Duchenne muscular dystrophy.

Dongsheng Duan, Nathalie Goemans, Shin'ichi Takeda, Eugenio Mercuri, Annemieke Aartsma-Rus

2 registry-linked trialsOpen access · bronzeAbstract readReview
In one paragraph

Review in Nature reviews. Disease primers, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 819 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
819citing papers in PubMed, 2 pooled it
64.3field-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.

NCT06682585 nacompletednot on this mapstarted 2024, after this paper: background citation

Determination of Nutritional Status of Duchenne Muscular Dystrophy Patients and Evaluation of the Effectiveness of Disease-Specific Nutrition Therapy

TypeinterventionalSponsorAnkara UniversityRan2024 to 2025Enrolled9ConditionsDuchenne Muscular Dystrophy (DMD)ArmsIndividualised dietary intervention
NCT07436143 narecruitingnot on this mapstarted 2025, after this paper: background citation

Effects of Alexander Technique on Posture Control of Upper Limb and Quality of Life in Children With Duchenne Muscular Dystrophy

TypeinterventionalSponsorRiphah International UniversityRan2025 to 2026Enrolled33ConditionsChild, OnlyArmsAlexander Technique Program
3 · Its place in the literature

Who cites it

819 citing papers in PubMed, 2 syntheses or guidelines pooled it, 1,197 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Trial
  4. Trial
  5. Trial
  6. Trial
  7. Article
  8. Article
  9. Review
  10. Review
  11. Article
  12. Article
  13. Article
  14. Article
  15. Article
  16. The histone deacetylase family in health and disease.Signal transduction and targeted therapy · 2026
    Review
  17. Preclinical efficacy of a gene therapy forMolecular therapy. Advances · 2026
    Article
  18. Article
  19. Article
  20. Review

759 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

5 authors at 5 institutions in 5 countries.

Dongsheng DuanDepartment of Molecular Microbiology and Immunology and Department of Neurology, School of Medicine; Department of Biomedical Sciences, College of Veterinary Medicine; Department of Biomedical, Biological & Chemical Engineering, College of Engineering, University of Missouri, Columbia, MO, USA.ORCID http://orcid.org/0000-0003-4109-1132
Nathalie GoemansDepartment of Child Neurology, University Hospitals Leuven, Leuven, Belgium.
Shin'ichi TakedaNational Center of Neurology and Psychiatry, Tokyo, Japan.ORCID http://orcid.org/0000-0003-4780-6290
Eugenio MercuriCentro Clinico Nemo, Policlinico Gemelli, Rome, Italy.
Annemieke Aartsma-RusDepartment of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands. a.m.aartsma-rus@lumc.nl.ORCID http://orcid.org/0000-0003-1565-654X
KU Leuven · BELeiden University Medical Center · NLMissouri College · USNational Center of Neurology and Psychiatry · JPUniversità Cattolica del Sacro Cuore · IT

Funding

Whole body single AAV microgene therapy in canine DMDR01NS090634 · NINDS · UNIVERSITY OF MISSOURI-COLUMBIA · PI Dongsheng Duan · 2015 to 2026
$4.2M
Treatment of Duchenne Muscular Dystrophy with the Muscle Calcium PumpR01AR070517 · NIAMS · UNIVERSITY OF MISSOURI-COLUMBIA · PI DUAN, DONGSHENG · 2016 to 2020
$3.1M
University Training Program in Biomolecular and Tissue EngineeringT32GM144291 · NIGMS · DUKE UNIVERSITY · PI Charles A. Gersbach, Tatiana Segura · 2022 to 2026
$2.6M
CRISPR/Cas9-Based Gene Editing for the Correction of Duchenne Muscular DystrophyR01AR069085 · NIAMS · DUKE UNIVERSITY · PI GERSBACH, CHARLES A. · 2016 to 2020
$1.7M
NIAMS NIH HHS R01 AR069085NIAMS NIH HHS R01 AR070517NIGMS NIH HHS T32 GM144291NINDS NIH HHS R01 NS090634
6 · The paper itself

Abstract

Duchenne muscular dystrophy is a severe, progressive, muscle-wasting disease that leads to difficulties with movement and, eventually, to the need for assisted ventilation and premature death. The disease is caused by mutations in DMD (encoding dystrophin) that abolish the production of dystrophin in muscle. Muscles without dystrophin are more sensitive to damage, resulting in progressive loss of muscle tissue and function, in addition to cardiomyopathy. Recent studies have greatly deepened our understanding of the primary and secondary pathogenetic mechanisms. Guidelines for the multidisciplinary care for Duchenne muscular dystrophy that address obtaining a genetic diagnosis and managing the various aspects of the disease have been established. In addition, a number of therapies that aim to restore the missing dystrophin protein or address secondary pathology have received regulatory approval and many others are in clinical development.

Indexed as

Muscular Dystrophy, DuchenneAnimalsDisease Models, AnimalDystrophinHumansDystrophin

Identifiers

PMID33602943
PMCPMC10557455
OpenAlexW3131782536

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

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.