Evidence map›Paper›PMID 42161251›Full record

ArticleJournal of neuromuscular diseases2026

Quantification of a serum titin fragment reflects dystrophin restoration in mdx mice and disease severity in patients with dystrophinopathies.

Camilla Johansson, Jessica F Boehler, Kristy J Brown, Albert Jiménez-Requena, Andreas Hober, Fredrik Edfors, Åsa Sivertsson, Gabriella Hober, Zaïda Koeks, Nienke M van de Velde and 5 more

Abstract read
PubMed Full text at PMC Publisher
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

15 authors.

Camilla JohanssonDepartment of Protein Science, School of Chemistry, Biotechnology and Health, KTH Royal Institute of Technology, Stockholm, Sweden.
Jessica F BoehlerSolid Biosciences Inc, Charlestown, MA, USA.
Kristy J BrownSomite Therapeutics, Brighton, MA, USA.
Albert Jiménez-RequenaDepartment of Protein Science, School of Chemistry, Biotechnology and Health, KTH Royal Institute of Technology, Stockholm, Sweden.
Andreas HoberScience for Life Laboratory, School of Chemistry, Biology and Health, KTH Royal Institute of Technology, Stockholm, Sweden.ORCID 0000-0001-8947-2562
Fredrik EdforsScience for Life Laboratory, School of Chemistry, Biology and Health, KTH Royal Institute of Technology, Stockholm, Sweden.
Åsa SivertssonScience for Life Laboratory, School of Chemistry, Biology and Health, KTH Royal Institute of Technology, Stockholm, Sweden.
Gabriella HoberDepartment of Protein Science, School of Chemistry, Biotechnology and Health, KTH Royal Institute of Technology, Stockholm, Sweden.
Zaïda KoeksDepartment of Neurology, Leiden University Medical Center, Leiden, The Netherlands.
Nienke M van de VeldeDepartment of Neurology, Leiden University Medical Center, Leiden, The Netherlands.
Mathias UhlénScience for Life Laboratory, School of Chemistry, Biology and Health, KTH Royal Institute of Technology, Stockholm, Sweden.
Hanna TegelDepartment of Protein Science, School of Chemistry, Biotechnology and Health, KTH Royal Institute of Technology, Stockholm, Sweden.
Erik H NiksDepartment of Neurology, Leiden University Medical Center, Leiden, The Netherlands.
Pietro SpitaliDepartment of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.
Cristina Al-Khalili SzigyartoDepartment of Protein Science, School of Chemistry, Biotechnology and Health, KTH Royal Institute of Technology, Stockholm, Sweden.

Funding

Biomarker Signatures for Duchenne Muscular DystrophyR61NS119639 · NINDS · STATE UNIVERSITY OF NY,BINGHAMTON · PI DANG, UTKARSH J, HATHOUT, YETRIB · 2022 to 2024
$1.6M
NINDS NIH HHS R61 NS119639
6 · The paper itself

Abstract

Increased dystrophin expression in muscle has been accepted as a surrogate endpoint for accelerated approval of gene therapies within Duchenne Muscular Dystrophy (DMD). Previous studies identified an N-terminal titin fragment in urine as a biomarker suitable for monitoring disease progression and therapeutic interventions. Plasma levels of a titin fragment comprising aa 2,229-2,352 were found to negatively correlate with dystrophin expression in

Indexed as

becker muscular dystrophyduchenne muscular dystrophymicro-dystrophin therapyprotein biomarkersandwich immunoassaytitin

Identifiers

PMID42161251
PMCPMC13438847

What OpenQuestion holds

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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.