SynthesisHuman genetics2025
Efficacy of delandistrogene moxeparvovec on Duchenne muscular dystrophy: a systematic review and meta-analysis.
Synthesis in Human genetics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
4 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Accuracy of the molecular diagnosis of duchenne and becker muscular dystrophy: A systematic review with meta-analysis.PloS one · 2026Pooled it
- Six-Minute Activity-95Pediatric neurology · 2026Observational
- Systematic analysis of the adverse effects of used clinical antisense oligonucleotide drugs in DMD patients based on the FAERS database.European journal of clinical pharmacology · 2026Article
- Engineering Targeted Gene Delivery Systems for Primary Hereditary Skeletal Myopathies: Current Strategies and Future Perspectives.Biomedicines · 2025Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
Abstract
Delandistrogene moxeparvovec was recently approved for Duchenne Muscular Dystrophy (DMD), using an adeno-associated virus to introduce a microdystrophin gene; however, the evidence is limited. The aim was to assess the effects of delandistrogene moxeparvovec on motor function and safety in the population with DMD. A systematic search of MEDLINE, Scopus, Web of Science and the Cochrane Library was conducted from their inception to May 2025. Clinical trials that assessed the effect or safety of delandistrogene moxeparvovec in the DMD population were included. For efficacy, the North Star Ambulatory Assessment (NSAA), Supine to Stand (TTSTAND), Climb 4 Stairs (TTCLIMB), 100-meter Timed Walk Test, and 10-meter Run Test were used, and for safety, the proportion of main adverse events was determined. Meta-analyses were performed for each outcome. Five trials involving 190 participants (4.8 to 6.0 years) were included. At one year, in the pre-post designs, there was an improvement of 2.63 points (95%CI: 1.74, 3.52) on the NSAA and − 0.29 s (95%CI: -0.52, -0.06) on the TTSTAND, while compared with the placebo, there was an improvement of -0.64 s (95%CI: -0.99, -0.30) in the TTSTAND. Moreover, the effect on some outcomes tended to be greater in participants aged 4–5 years. Severe side effects occurred in 10–15% of the samples. At one year after treatment, delandistrogene moxeparvovec had a modest effect on the progression of DMD. It is expected that future studies will confirm this efficacy, particularly in longer trials, as it is difficult to recover lost or ungained motor function.
Indexed as
Identifiers
40569438What Socratic 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 Socratic graph.