Trial reportAnnals of neurology2016
Multicenter prospective longitudinal study of magnetic resonance biomarkers in a large duchenne muscular dystrophy cohort.
Trial report in Annals of neurology, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 3 registered trials, which are not on this map. Cited by 102 papers, 1 of them a synthesis 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.
GRASP-01-003: Trial Readiness and Endpoint Assessment in LGMD R1
Long-Term Development of Muscular Dystrophy Outcome Assessments (GRASP-01-005)
Vasodilators and Exercise as Adjuvant Therapy for Duchenne Muscular Dystrophy (VASO-REx Study)
Who cites it
102 citing papers in PubMed, 1 synthesis or guideline pooled it, 162 citations in OpenAlex.
- Using MRI to quantify skeletal muscle pathology in Duchenne muscular dystrophy: A systematic mapping review.Muscle & nerve · 2021Pooled it
- Dual-energy X-ray absorptiometry measures of lean body mass as a biomarker for progression in boys with Duchenne muscular dystrophy.Scientific reports · 2022Trial
- Upper limb disease evolution in exon 53 skipping eligible patients with Duchenne muscular dystrophy.Annals of clinical and translational neurology · 2021Trial
- Quantifying disease activity in fatty-infiltrated skeletal muscle by IDEAL-CPMG in Duchenne muscular dystrophy.Neuromuscular disorders : NMD · 2016Trial
- Observational
- Total body and appendicular lean mass by dual-energy X-ray absorptiometry are highly associated with muscle function and bone mineral density in Becker muscular dystrophy.Journal of neuromuscular diseases · 2026Article
- Myocardial fat fraction in Becker muscular dystrophy and women carrying pathogenicJournal of neuromuscular diseases · 2026Article
- Elbow Flexors Muscle Fat Fraction Is a Sensitive and Relevant Outcome Measure in Nonambulant Patients With DMD.NMR in biomedicine · 2026Article
- Myasthenia gravis: from recognition of heterogeneity to a paradigm shift toward precision therapy.Frontiers in immunology · 2026Review
- Intramuscular adipose tissue restricts functional muscle recovery.Cell reports · 2025Article
- Multi-scale machine learning model predicts muscle and functional disease progression.Scientific reports · 2025Article
- Multiparametric quantitative magnetic resonance imaging of skeletal muscle in CKD.American journal of physiology. Renal physiology · 2025Article
- Longitudinal Follow-Up of Patients With Duchenne Muscular Dystrophy Using QuantitativeJournal of cachexia, sarcopenia and muscle · 2025Article
- Physical Activity Correlates With Skeletal Muscle MRI Findings in Individuals With Duchenne Muscular Dystrophy.Muscle & nerve · 2025Article
- Multi-parametric quantitative MRI of the lower limb muscles in a longitudinal study of limb-girdle muscular dystrophy R9.PloS one · 2025Article
- Serum protein and imaging biomarkers after intermittent steroid treatment in muscular dystrophy.Scientific reports · 2024Article
- Skeletal muscle symptoms and quantitative MRI in females with dystrophinopathy.Muscle & nerve · 2024Article
- Serum protein and imaging biomarkers after intermittent steroid treatment in muscular dystrophy.medRxiv : the preprint server for health sciences · 2024Article
- Diffusion tensor imaging reveals subclinical alterations in muscles of patients with Becker muscular dystrophy.The British journal of radiology · 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
42 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
17 authors at 7 institutions in 1 country.
Funding
Abstract
objectiveThe aim of this study was to describe Duchenne muscular dystrophy (DMD) disease progression in the lower extremity muscles over 12 months using quantitative magnetic resonance (MR) biomarkers, collected across three sites in a large cohort.
methodsA total of 109 ambulatory boys with DMD (8.7 ± 2.0 years; range, 5.0-12.9) completed baseline and 1-year follow-up quantitative MR imaging (transverse relaxation time constant; MRI-T2 ), MR spectroscopy (fat fraction and (1) H2 O T2 ), and 6-minute walk test (6MWT) measurements. A subset of boys completed additional measurements after 3 or 6 months.
resultsMRI-T2 and fat fraction increased significantly over 12 months in all age groups, including in 5- to 6.9-year-old boys. Significant increases in vastus lateralis (VL) fat fraction were observed in 3 and 6 months. Even in boys whose 6MWT performance improved or remained stable over 1 year, significant increases in MRI-T2 and fat fraction were found. Of all the muscles examined, the VL and biceps femoris long head were the most responsive to disease progression in boys with DMD.
interpretationMR biomarkers are responsive to disease progression in 5- to 12.9-year-old boys with DMD and able to detect subclinical disease progression in DMD, even within short (3-6 months) time periods. The measured sensitivity of MR biomarkers in this multicenter study may be critically important to future clinical trials, allowing for smaller sample sizes and/or shorter study windows in this fatal rare disease.
Indexed as
Identifiers
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.