Evidence map›Paper›PMID 42287226›Full record

ArticleJournal of neuromuscular diseases2026

Meeting report: 2025 muscular dystrophy association summit on 'safety and challenges in gene therapy of neuromuscular diseases'.

Brian C Lin, Angela Lek, Carsten G Bönnemann, Barry J Byrne

Abstract read
In one paragraph

Article in Journal of neuromuscular diseases, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the 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.

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

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

4 authors.

Brian C LinMuscular Dystrophy Association, Chicago, IL, USA.
Angela LekMuscular Dystrophy Association, Chicago, IL, USA.
Carsten G BönnemannDepartment of Genomic and Translational Neuroscience, St. Jude Children's Research Hospital, Memphis, TN, USA.
Barry J ByrneDepartment of Pediatrics and Powell Gene Therapy Center, University of Florida, Gainesville, FL, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The 2025 MDA Gene Therapy Summit brought together leading researchers, clinicians, and industry representatives to discuss the latest advancements and challenges in the development and clinical translation of genetic therapies for neuromuscular disorders. The meeting featured in-depth presentations on clinical trials, therapeutic developments, and regulatory pathways for AAV-mediated gene therapies, highlighting both successes and challenges in the field.

Indexed as

AAVgene therapymuscular dystrophyneuromuscular disease

Identifiers

PMID42287226
PMCPMC13438967

What OpenQuestion holds

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Registered trials

None linked

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.