Evidence map›Paper›PMID 42600180›Full record

ArticleJournal of neuromuscular diseases2026

Clinical, histopathological, and biomarker characterization of XLMTM and ADCNM: Operational lessons, screening and baseline data of the Unite-CNM study.

Olga Prikhodko, Tatyana A Vetter, Sophie Colombo, Chris Freitag, Dominique Nijkamp, Louise Eyler, Leen Thielemans, Jonathan Baets, Mads Stemmerik, John Vissing and 11 more

Registry-linked trialAbstract 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. It is linked to trial NCT04033159 (A Phase 1/2 Trial on the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Exploratory Efficacy of DYN101 in Patients ≥ 16 Years of Age With Centronuclear Myopathies Caused by Mutations in DNM2 or MTM1.), 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.

NCT04033159 phase1 / phase2terminatednot on this map

A Phase 1/2 Trial on the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Exploratory Efficacy of DYN101 in Patients ≥ 16 Years of Age With Centronuclear Myopathies Caused by Mutations in DNM2 or MTM1.

TypeinterventionalSponsorDynacureRan2020 to 2022Enrolled14ConditionsCentronuclear MyopathyArmsDYN101
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

21 authors.

Olga PrikhodkoIonis Pharmaceuticals, Carlsbad, CA, Essen, USA.
Tatyana A VetterDiverge Translational Science Laboratory, Milwaukee, WI, USA.
Sophie ColomboDynacure SA (now Flamingo Therapeutics NV), Louvain, Belgium.
Chris FreitagDynacure SA (now Flamingo Therapeutics NV), Louvain, Belgium.
Dominique NijkampDynacure SA (now Flamingo Therapeutics NV), Louvain, Belgium.
Louise EylerDynacure SA (now Flamingo Therapeutics NV), Louvain, Belgium.
Leen ThielemansDynacure SA (now Flamingo Therapeutics NV), Louvain, Belgium.
Jonathan BaetsTranslational Neurosciences, Faculty of Medicine and Health Sciences, University of Antwerp, Antwerp, Belgium.
Mads StemmerikCopenhagen Neuromuscular Center, Department of Neurology, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark.
John VissingCopenhagen Neuromuscular Center, Department of Neurology, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark.
Ros QuinlivanCentre for Neuromuscular Diseases, UCL Institute of Neurology, National Hospital for Neurology and Neurosurgery, London, UK.
Michela GuglieriJohn Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
Federica MontagneseFriedrich-Baur-Institut, Department of Neurology, Ludwig Maximilian University (LMU), Munich, Germany.
Benedikt SchoserFriedrich-Baur-Institut, Department of Neurology, Ludwig Maximilian University (LMU), Munich, Germany.
Frederik BraunDepartment of Pediatric Neurology, Centre for Neuromuscular Disorders, Center for Translational Neuro and Behavioral Sciences, University Duisburg-Essen, Germany.ORCID 0000-0002-3601-2746
Ulrike ScharaDepartment of Pediatric Neurology, Centre for Neuromuscular Disorders, Center for Translational Neuro and Behavioral Sciences, University Duisburg-Essen, Germany.
Kris E LeeuwenbergDepartment of Neurology, Donders Institute for Brain, Cognition and Behavior, Radboud University Medical Center, Nijmegen, The Netherlands.
Nens van AlfenDepartment of Neurophysiology and Department of Neurology, Donders Center for Cognitive Neuroimaging, Radboud University Medical Center, Nijmegen, The Netherlands.
Michael W LawlorDiverge Translational Science Laboratory, Milwaukee, WI, USA.
Belinda S CowlingIonis Pharmaceuticals, Carlsbad, CA, Essen, USA.
Nicol C VoermansDepartment of Neurology, Donders Institute for Brain, Cognition and Behavior, Radboud University Medical Center, Nijmegen, The Netherlands.ORCID 0000-0002-5837-7295

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

BackgroundX-linked myotubular myopathy (XLMTM) and autosomal dominant centronuclear myopathy (ADCNM) are rare neuromuscular disorders characterized by severe weakness and respiratory impairment and have recently been the focus of therapeutic development. Robust baseline data are essential to understand disease trajectories and enable trial readiness. We present baseline clinical, functional, and biomarker findings from the terminated Unite-CNM trial (NCT04033159), a basket study of the antisense oligonucleotide DYN101, designed to modulate

Indexed as

centronuclear myopathyclinical trialfreezing artifactgoal attainment scalehistopathology analysisimage analysismuscle biopsyultrasoundX-linked myotubular myopathy

Identifiers

PMID42600180
PMCPMC13476381

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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.