Evidence map›Paper›PMID 42384320›Full record

ArticleCardiology and therapy2026

Cardiac Safety Outcomes in Delandistrogene Moxeparvovec Clinical Trials for Duchenne Muscular Dystrophy with Up to 5 Years of Follow-up.

Aravindhan Veerapandiyan, John Bourke, Jonathan H Soslow, John W Day, Craig M McDonald, Jerry R Mendell, Craig M Zaidman, Stefanie Mason, Jianfeng Meng, Mark Vivien and 3 more

4 registry-linked trialsAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Cardiology and therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 4 registered trials, which are not on this map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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.

NCT03375164 phase1 / phase2completednot on this map

Systemic Gene Delivery Phase I/IIa Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MHCK7.Micro-dystrophin (microDys-IV-001)

TypeinterventionalSponsorSarepta Therapeutics, Inc.Ran2018 to 2023Enrolled4ConditionsDuchenne Muscular DystrophyArmsdelandistrogene moxeparvovec
NCT03769116 phase1 / phase2completednot on this map

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial for Duchenne Muscular Dystrophy Using SRP-9001

TypeinterventionalSponsorSarepta Therapeutics, Inc.Ran2018 to 2023Enrolled41ConditionsMuscular Dystrophy, DuchenneArmsdelandistrogene moxeparvovec, placebo
NCT04626674 phase1recruitingnot on this map

An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects With Duchenne Muscular Dystrophy (ENDEAVOR)

TypeinterventionalSponsorSarepta Therapeutics, Inc.Ran2020 to 2028Enrolled83ConditionsDuchenne Muscular DystrophyArmsdelandistrogene moxeparvovec
NCT05096221 phase3completednot on this map

A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK)

TypeinterventionalSponsorSarepta Therapeutics, Inc.Ran2021 to 2024Enrolled126ConditionsDuchenne Muscular DystrophyArmsdelandistrogene moxeparvovec, placebo
3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Review
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

13 authors.

Aravindhan VeerapandiyanUniversity of Arkansas for Medical Sciences, Arkansas Children's Hospital, Little Rock, AR, USA.ORCID http://orcid.org/0000-0002-3065-3956
John BourkeNUTH NHS Hospitals Foundation Trust, Newcastle upon Tyne, UK.ORCID http://orcid.org/0000-0001-7857-9073
Jonathan H SoslowVanderbilt University Medical Center, Nashville, TN, USA.ORCID http://orcid.org/0000-0001-9194-5330
John W DayStanford University, Palo Alto, CA, USA.ORCID http://orcid.org/0000-0002-0086-9529
Craig M McDonaldUniversity of California Davis Health, Sacramento, CA, USA.ORCID http://orcid.org/0000-0002-8779-3220
Jerry R MendellSarepta Therapeutics, Inc., 215 First St, Cambridge, MA, 02142, USA.ORCID http://orcid.org/0009-0005-5269-4467
Craig M ZaidmanWashington University in Saint Louis, St. Louis, MO, USA.ORCID http://orcid.org/0000-0002-1255-5877
Stefanie MasonSarepta Therapeutics, Inc., 215 First St, Cambridge, MA, 02142, USA.ORCID http://orcid.org/0000-0002-7649-9026
Jianfeng MengSarepta Therapeutics, Inc., 215 First St, Cambridge, MA, 02142, USA.ORCID http://orcid.org/0009-0006-8809-4305
Mark VivienSarepta Therapeutics, Inc., 215 First St, Cambridge, MA, 02142, USA.ORCID http://orcid.org/0009-0009-6552-9152
Alexander P MurphyRoche Products Ltd, Welwyn Garden City, UK.
Christoph WandelF. Hoffmann-La Roche Ltd, Basel, Switzerland.ORCID http://orcid.org/0009-0005-6303-8289
James RichardsonSarepta Therapeutics, Inc., 215 First St, Cambridge, MA, 02142, USA. jrichardson@sarepta.com.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionIn patients with Duchenne muscular dystrophy (DMD), the gene transfer therapy delandistrogene moxeparvovec delivers a functional form of dystrophin, which has been shown to stabilize or slow disease progression.

methodsWe assessed cardiac safety of delandistrogene moxeparvovec in clinical trials with ≤ 5 years of follow-up. Data were collected from clinical trials 101 (NCT03375164, n = 4), 102 (NCT03769116, n = 41), ENDEAVOR (NCT04626674, n = 48), and EMBARK (NCT05096221, n = 125), which excluded patients with left ventricular ejection fraction (LVEF) < 40%. Adverse events and cardiac echocardiography were assessed regularly in all trials. Troponin I was assessed regularly in ENDEAVOR and EMBARK. Cardiac magnetic resonance imaging (MRI; without gadolinium enhancement) was assessed within an EMBARK substudy.

resultsOf 218 patients (baseline mean age [range], 6.4 [3.2-20.2] years; mean LVEF [range], 63.8% [48.9-78.0%]), 210 (96%) were ambulatory; 216 received delandistrogene moxeparvovec treatment. Two myocarditis cases were reported within 4 days after delandistrogene moxeparvovec infusion; both resolved within 3 weeks. Except in the two myocarditis cases, troponin I fluctuations were asymptomatic. Thirteen patients with baseline and postbaseline echocardiography data had elevated troponin I at baseline; 1 year post infusion, only one of these patients had LVEF < 50%. LVEF in all four patients with 5-year follow-up remained > 50%. Although cardiac MRI without gadolinium revealed no relevant differences in heart function between patients 1 or 2 years after delandistrogene moxeparvovec versus patients 1 year after placebo infusion, subclinical fibrosis cannot be ruled out.

conclusionResults from delandistrogene moxeparvovec trials with 1 to 5 years of follow-up suggest a manageable cardiac safety profile in this study population of predominantly younger, ambulatory patients with DMD who had no signs of persistent treatment-related cardiac injury.

Indexed as

Adverse cardiac eventDrug-related side effects and adverse reactionsGene therapy agentsGenetic therapyMuscular dystrophy, Duchenne

Identifiers

PMID42384320
PMCPMC13542014

What OpenQuestion holds

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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.