ReviewNature reviews. Neurology2019
Therapeutic developments for Duchenne muscular dystrophy.
Review in Nature reviews. Neurology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06290713 (Vasodilators and Exercise as Adjuvant Therapy for Duchenne Muscular Dystrophy), which is not on this map. Cited by 241 papers, 3 of them syntheses that pooled 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.
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.
Vasodilators and Exercise as Adjuvant Therapy for Duchenne Muscular Dystrophy (VASO-REx Study)
Who cites it
241 citing papers in PubMed, 3 syntheses or guidelines pooled it, 379 citations in OpenAlex.
- Meta-analysis of the efficacy and safety of vamorolone in Duchenne muscular dystrophy.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2025Pooled it
- Efficacy and safety of different doses of vamorolone in boys with Duchenne muscular dystrophy: a systematic review and network meta-analysis.Frontiers in neurology · 2024Pooled it
- Dilated cardiomyopathy as the initial presentation of Becker muscular dystrophy: a systematic review of published cases.Orphanet journal of rare diseases · 2022Pooled it
- Effects of Bisphosphonates on Bone Micro-Architecture of Children With Duchenne Muscular Dystrophy: A Prospective Comparative Study.Journal of cachexia, sarcopenia and muscle · 2026Trial
- Safety and efficacy of viltolarsen in ambulatory and nonambulatory males with Duchenne muscular dystrophy.Scientific reports · 2024Trial
- Validity of remote live stream video evaluation of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy.PloS one · 2024Trial
- Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study.Journal of neuromuscular diseases · 2023Trial
- Trial
- Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy.Journal of neuromuscular diseases · 2022Trial
- A Registry-Based Perspective of Interventional Clinical Trials for Duchenne Muscular Dystrophy.Cureus · 2026Article
- Characterization of Dystrophin-Related Syndromes: Carriers, DMD, and BMD.Muscles (Basel, Switzerland) · 2026Review
- Engineered exosomes improve myocardial cell membrane integrity and heart function in dystrophic cardiomyopathy.Clinical and translational medicine · 2026Article
- 2nd NMD4C basic research summer school on promoting standardized protocols to advance translational research in neuromuscular disorders.Journal of neuromuscular diseases · 2026Article
- Skeletal-muscle-targeted non-viral delivery of full-length DMD mRNA for Duchenne muscular dystrophy.Nature biomedical engineering · 2026Article
- Analysis of adverse event reporting with casimersen: a pharmacovigilance study based on the United States food and drug administration adverse event reporting system database.International journal of clinical pharmacy · 2026Article
- ANXA11 suppression restores muscular function in the mdx mouse model of Duchenne muscular dystrophy (DMD).Nature communications · 2026Article
- Safety and Tolerability of Givinostat: Evidence From Real-World and Clinical Practice.Annals of clinical and translational neurology · 2026Article
- Molecular Mechanisms and Therapeutic Strategies in Heart Failure Due to Dystrophin Deficiency: A Comprehensive Review.Reviews in cardiovascular medicine · 2026Review
- Transcytotic transportation of size-controlled nanocarriers into dystrophic skeletal muscle leads to therapeutic outcome in mice.Nature communications · 2025Article
- Radiomics with Ultrasound Radiofrequency Data for Improving Evaluation of Duchenne Muscular Dystrophy.Journal of imaging informatics in medicine · 2025Article
181 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Duchenne muscular dystrophy (DMD) is caused by the lack of functional dystrophin protein. Improvements in patient care and disease management have slowed down disease progression, but current treatments cannot stop the relentless loss of muscle tissue and function, which leads to premature death. Research is ongoing to develop effective therapies for DMD. Gene-addition, exon-skipping, stop codon readthrough and genome-editing therapies can restore the expression of partially functional dystrophin protein, whereas other therapeutic approaches aim to improve muscle function and quality by targeting pathways involved in the pathogenesis of DMD. This Review outlines important developments in these research areas and specifically focuses on new therapies that are in the clinical trial phase or have already been approved.
Indexed as
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What OpenQuestion holds
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.