ArticlePloS one2016
Discovery of Metabolic Biomarkers for Duchenne Muscular Dystrophy within a Natural History Study.
Article in PloS one, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 28 papers.
What it found
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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.
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Who cites it
28 citing papers in PubMed, 43 citations in OpenAlex.
- Creatine/Creatinine Ratio and Myostatin as Biomarkers to Monitor Muscle Function in Duchenne Muscular Dystrophy Patients.Journal of cachexia, sarcopenia and muscle · 2026Observational
- A natural history study of Chinese individuals with Duchenne muscular dystrophy: Results from 2 years of follow-up and beyond.PloS one · 2026Observational
- Data from multi-national aging cohorts show dysregulated metabolic pathways in people with physio-cognitive decline.Communications medicine · 2025Article
- D- and L-Amino Acid Blood Concentrations Are Affected in Children With Duchenne Muscular Dystrophy.Journal of cellular and molecular medicine · 2025Article
- Cerebrospinal fluid metabolomics, lipidomics and serine pathway dysfunction in myalgic encephalomyelitis/chronic fatigue syndroome (ME/CFS).Scientific reports · 2025Article
- Exertional Exhaustion (Post-Exertional Malaise, PEM) Evaluated by the Effects of Exercise on Cerebrospinal Fluid Metabolomics-Lipidomics and Serine Pathway in Myalgic Encephalomyelitis/Chronic Fatigue Syndrome.International journal of molecular sciences · 2025Article
- Characterization of disease-specific alterations in metabolites and effects of mesenchymal stromal cells on dystrophic muscles.Frontiers in cell and developmental biology · 2024Article
- Serum metabolomic signatures of patients with rare neurogenetic diseases: an insight into potential biomarkers and treatment targets.Frontiers in molecular neuroscience · 2024Article
- Evaluation of a six-minute walk test in the DE50-MD canine model of Duchenne muscular dystrophy and its effect on blood-borne biomarkers.Wellcome open research · 2024Article
- Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies - Developing Potential Treatments for the Entire Spectrum of Disease.Journal of neuromuscular diseases · 2024Article
- The metabolomic plasma profile of patients with Duchenne muscular dystrophy: providing new evidence for its pathogenesis.Orphanet journal of rare diseases · 2023Article
- Orthogonal proteomics methods warrant the development of Duchenne muscular dystrophy biomarkers.Clinical proteomics · 2023Article
- Longitudinal Assessment of Creatine Kinase, Creatine/CreatinineNeurology · 2023Article
- Serum inflammatory cytokines as disease biomarkers in the DE50-MD dog model of Duchenne muscular dystrophy.Disease models & mechanisms · 2022Article
- Biomarkers in Duchenne Muscular Dystrophy.Current heart failure reports · 2022Review
- Serum creatinine as a biomarker for dystrophinopathy: a cross-sectional and longitudinal study.BMC neurology · 2021Article
- Pathway testing for longitudinal metabolomics.Statistics in medicine · 2021Article
- Article
- Circulating Biomarkers in Muscular Dystrophies: Disease and Therapy Monitoring.Molecular therapy. Methods & clinical development · 2020Review
- Disease-specific and glucocorticoid-responsive serum biomarkers for Duchenne Muscular Dystrophy.Scientific reports · 2019Article
Corrections and comments
- Erratum issued
Authors and funding
15 authors at 5 institutions in 2 countries.
Funding
Abstract
Serum metabolite profiling in Duchenne muscular dystrophy (DMD) may enable discovery of valuable molecular markers for disease progression and treatment response. Serum samples from 51 DMD patients from a natural history study and 22 age-matched healthy volunteers were profiled using liquid chromatography coupled to mass spectrometry (LC-MS) for discovery of novel circulating serum metabolites associated with DMD. Fourteen metabolites were found significantly altered (1% false discovery rate) in their levels between DMD patients and healthy controls while adjusting for age and study site and allowing for an interaction between disease status and age. Increased metabolites included arginine, creatine and unknown compounds at m/z of 357 and 312 while decreased metabolites included creatinine, androgen derivatives and other unknown yet to be identified compounds. Furthermore, the creatine to creatinine ratio is significantly associated with disease progression in DMD patients. This ratio sharply increased with age in DMD patients while it decreased with age in healthy controls. Overall, this study yielded promising metabolic signatures that could prove useful to monitor DMD disease progression and response to therapies in the future.
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Registered trials
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