Evidence map›Paper›PMID 41047627›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2026

Real-world outcomes of delandistrogene moxeparvovec gene therapy: Motor outcomes and emerging safety concerns.

Dennis J Keselman, Juliana C Small, Tiffany Seneviratne, Sarah McCague, Taylor L Kaschak, Sabrina W Yum, Andrea O'Brien, John F Brandsema, Tamir Diamond, Kathleen M Loomes and 8 more

Abstract read
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In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
–field-weighted citation impact
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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

4 citing papers in PubMed.

  1. [Duchenne muscular dystrophy-associated cardiomyopathy: advances in diagnosis and management].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2026
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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

18 authors.

Dennis J KeselmanDivision of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Neurology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
Juliana C SmallClinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Tiffany SeneviratneClinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Sarah McCagueClinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Taylor L KaschakDivision of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Sabrina W YumDivision of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Neurology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
Andrea O'BrienDivision of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
John F BrandsemaDivision of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Neurology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
Tamir DiamondBioinformatics and Division of Gastroenterology, Hepatology and Nutrition, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
Kathleen M LoomesBioinformatics and Division of Gastroenterology, Hepatology and Nutrition, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
Kimberly Y LinDepartment of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA; Division of Cardiology, Cardiac Center, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Carol A Wittlieb-WeberDepartment of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA; Division of Cardiology, Cardiac Center, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Oscar H MayerDepartment of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA; Division of Pulmonology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Scott G DanielBioinformatics and Division of Gastroenterology, Hepatology and Nutrition, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Amy T WaldmanDivision of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Neurology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Benjamin J Samelson-JonesClinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA; Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Susan E MatesanzDivision of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Neurology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA; Clinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA. Electronic address: matesanzs@chop.edu.
Lindsey A GeorgeClinical In Vivo Gene Therapy, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA; Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA. Electronic address: georgel@chop.edu.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Delandistrogene moxeparvovec is currently the only commercially approved gene therapy for Duchenne muscular dystrophy (DMD). Herein, we report real-world data of 11 ambulatory patients with DMD, ages 4-6, treated with commercial delandistrogene moxeparvovec. Patients were prospectively and uniformly monitored for 1 year post-gene transfer for safety and motor outcomes. Nine patients experienced 15 treatment-related toxicities, and 4 required escalation of corticosteroids. Side effects included gastrointestinal symptoms (n = 7), liver enzyme abnormalities (n = 4), acute liver injury (n = 2), and troponin I elevations (n = 3). In both patients with acute liver injury, troponin I elevations occurred in close temporal association beginning 8-9 weeks post-gene transfer and were responsive to corticosteroids. The clinical courses of these two patients were at least partially consistent with a cellular immune response to the adeno-associated viral vector (AAV) capsid. Troponin I elevations were asymptomatic and without acute functional changes on echocardiogram. The cohort had improvements in year 1 motor function assessments relative to baseline, including a statistically significant median 4-point increase in North Star Ambulatory Assessment score; however, important confounding factors, e.g., baseline corticosteroid use, limit interpretation and will be important to control for in future real-world datasets. Additional follow-up is required to determine long-term safety and motor outcomes, with unclear generalizability of our results.

Indexed as

DependovirusGenetic TherapyGenetic VectorsMuscular Dystrophy, DuchenneBiological ProductsChildChild, PreschoolFemaleHumansMaleRecombinant Fusion ProteinsTreatment OutcomeBiological ProductsRecombinant Fusion ProteinsZolgensmaAAVdelandistrogene moxeparvovecDMDreal-world outcomes

Identifiers

PMID41047627
PMCPMC12925777

What OpenQuestion holds

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LicenceCC BY-NC-ND
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Registered trials

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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.