Evidence map›Paper›PMID 42237476›Full record

Trial reportMuscle & nerve2026

Five-Year Outcomes With Delandistrogene Moxeparvovec in Patients With Duchenne Muscular Dystrophy: A Phase 1/2a Study.

Jerry R Mendell, Zarife Sahenk, Linda P Lowes, Megan A Iammarino, Lindsay N Alfano, Craig M McDonald, James E Signorovitch, Jim Jin, Jing Li, Stefanie Mason and 1 more

Registry-linked trialAbstract readClinical Trial, Phase IIClinical Trial, Phase I
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Muscle & nerve, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03375164 (Systemic Gene Delivery Phase I/IIa Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MHCK7.Micro-dystrophin), which is not on this map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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
3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

11 authors.

Jerry R MendellJerry Mendell Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.ORCID https://orcid.org/0000-0002-2691-0200
Zarife SahenkJerry Mendell Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.ORCID https://orcid.org/0000-0003-2974-3943
Linda P LowesJerry Mendell Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.ORCID https://orcid.org/0000-0003-4206-0557
Megan A IammarinoJerry Mendell Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.ORCID https://orcid.org/0000-0003-2800-3855
Lindsay N AlfanoJerry Mendell Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio, USA.ORCID https://orcid.org/0000-0002-2263-7569
Craig M McDonaldDepartments of Physical Medicine & Rehabilitation and Pediatrics, University of California Davis Health, Sacramento, California, USA.ORCID https://orcid.org/0000-0002-8779-3220
James E SignorovitchAnalysis Group Inc, Boston, Massachusetts, USA.ORCID https://orcid.org/0000-0002-4067-8962
Jim JinSarepta Therapeutics Inc., Cambridge, Massachusetts, USA.
Jing LiSarepta Therapeutics Inc., Cambridge, Massachusetts, USA.
Stefanie MasonSarepta Therapeutics Inc., Cambridge, Massachusetts, USA.ORCID https://orcid.org/0000-0002-7649-9026
Louise R Rodino-KlapacSarepta Therapeutics Inc., Cambridge, Massachusetts, USA.ORCID https://orcid.org/0000-0002-8966-5177

Funding

Nationwide Children's FoundationParent Project Muscular DystrophySarepta Therapeutics Inc.
6 · The paper itself

Abstract

aimsWe report 5-year results from a phase 1/2a study of delandistrogene moxeparvovec, a recombinant adeno-associated virus serotype rh74 vector-based gene therapy for Duchenne muscular dystrophy (DMD), with post hoc analyses contextualizing functional outcomes.

methodsFour ambulatory patients with DMD (≥ 4-< 8 years at enrollment) entered an open-label trial (Study 101; NCT03375164), receiving a single intravenous dose of delandistrogene moxeparvovec (2.0 × 10

resultsNo new safety signals were reported 5 years post-infusion. One patient had cardiomyopathy at year 5; however, this event was deemed unrelated to treatment. All delandistrogene moxeparvovec-treated patients (mean age, 10.2 years) remained ambulant. The 5-year NSAA total score mean change from baseline (standard deviation) for treated patients versus ECs was +7.5 (2.4) versus -3.9 (2.9) (least-squares mean between-group difference [standard error]: 9.8 [3.5], p = 0.0127). TTR and 10MWR mean times were stable, representing clinically meaningful differences versus ECs. An increased divergence in NSAA total score from 5-year natural history predictions favoring gene therapy was also seen. DISCUSSION: Findings support the long-term, manageable safety profile of delandistrogene moxeparvovec in ambulatory patients with appropriate monitoring and demonstrate stabilization or delayed disease progression with treatment compared with matched untreated ECs.

Indexed as

Genetic TherapyMuscular Dystrophy, DuchenneRecombinant Fusion ProteinsChildChild, PreschoolDependovirusGene Therapy AgentsHumansMaleTreatment Outcomedelandistrogene moxeparvovecRecombinant Fusion Proteinsdelandistrogene moxeparvovecDuchenne muscular dystrophygene therapymicro‐dystrophinrAAVrh74

Identifiers

PMID42237476
PMCPMC13332556

What Socratic holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

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.