Evidence map›Paper›PMID 39245687›Full record

Trial reportNature communications2024

Non-invasive optoacoustic imaging of glycogen-storage and muscle degeneration in late-onset Pompe disease.

Lina Tan, Jana Zschüntzsch, Stefanie Meyer, Alica Stobbe, Hannah Bruex, Adrian P Regensburger, Merle Claßen, Frauke Alves, Jörg Jüngert, Ulrich Rother and 16 more

Registry-linked trialAbstract readClinical Trial
In one paragraph

Trial report in Nature communications, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05083806 (Multispectral Optoacoustic Tomography for Translational Molecular Imaging in Pompe Disease), which is not on this map. Cited by 13 papers.

0numbers the graph read from it
0cells of the map it votes in
13citing 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.

NCT05083806 nacompletednot on this map

Multispectral Optoacoustic Tomography for Translational Molecular Imaging in Pompe Disease

TypeinterventionalSponsorUniversity of Erlangen-Nürnberg Medical SchoolRan2022 to 2023Enrolled20ConditionsPompe Disease, Pompe Disease (Late-onset), Pompe's Disease Juvenile Onset, Pompe Disease Infantile-OnsetArmsMultispectral Optoacoustic Tomography
3 · Its place in the literature

Who cites it

13 citing papers in PubMed.

  1. Article
  2. Article
  3. Article
  4. Optoacoustic muscle imaging.Journal of neuromuscular diseases · 2026
    Review
  5. Review
  6. Article
  7. Article
  8. Optical and Photoacoustic Imaging.Recent results in cancer research. Fortschritte der Krebsforschung. Progres dans les recherches sur le cancer · 2026
    Review
  9. Review
  10. Article
  11. Article
  12. Article
  13. Article
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

26 authors.

Lina TanDepartment of Pediatrics and Adolescent Medicine, University Hospital Erlangen, Friedrich-Alexander-Universität (FAU) Erlangen-Nürnberg, Erlangen, 91054, Germany.
Jana ZschüntzschNeuromuscular Disease Research, Clinic for Neurology, University Medical Center Göttingen (UMG), Göttingen, 37075, Germany.ORCID 0000-0002-7062-5319
Stefanie MeyerNeuromuscular Disease Research, Clinic for Neurology, University Medical Center Göttingen (UMG), Göttingen, 37075, Germany.
Alica StobbeNeuromuscular Disease Research, Clinic for Neurology, University Medical Center Göttingen (UMG), Göttingen, 37075, Germany.
Hannah BruexNeuromuscular Disease Research, Clinic for Neurology, University Medical Center Göttingen (UMG), Göttingen, 37075, Germany.
Adrian P RegensburgerDepartment of Pediatrics and Adolescent Medicine, University Hospital Erlangen, Friedrich-Alexander-Universität (FAU) Erlangen-Nürnberg, Erlangen, 91054, Germany.ORCID 0000-0001-8244-1261
Merle ClaßenDepartment of Pediatrics and Adolescent Medicine, University Hospital Erlangen, Friedrich-Alexander-Universität (FAU) Erlangen-Nürnberg, Erlangen, 91054, Germany.
Frauke AlvesTranslational Molecular Imaging, Max-Planck Institute for Multidisciplinary Sciences (MPI-NAT), City Campus, Göttingen, 37075, Germany.ORCID 0000-0002-6258-5409
Jörg JüngertDepartment of Pediatrics and Adolescent Medicine, University Hospital Erlangen, Friedrich-Alexander-Universität (FAU) Erlangen-Nürnberg, Erlangen, 91054, Germany.
Ulrich RotherDepartment of Vascular Surgery, University Hospital Erlangen, Friedrich-Alexander-Universität (FAU) Erlangen-Nürnberg, Erlangen, 91054, Germany.
Yi LiDepartment of Vascular Surgery, University Hospital Erlangen, Friedrich-Alexander-Universität (FAU) Erlangen-Nürnberg, Erlangen, 91054, Germany.ORCID 0000-0001-8385-4711
Vera DankoDepartment of Pediatrics and Adolescent Medicine, University Hospital Erlangen, Friedrich-Alexander-Universität (FAU) Erlangen-Nürnberg, Erlangen, 91054, Germany.
Werner LangDepartment of Vascular Surgery, University Hospital Erlangen, Friedrich-Alexander-Universität (FAU) Erlangen-Nürnberg, Erlangen, 91054, Germany.ORCID 0000-0003-4114-7589
Matthias TürkDepartment of Neurology, University Hospital Erlangen, Friedrich-Alexander-Universität (FAU) Erlangen-Nürnberg, Erlangen, 91054, Germany.
Sandy SchmidtInstitute of Radiology, University Hospital Erlangen, Friedrich-Alexander-Universität (FAU) Erlangen-Nürnberg, Erlangen, 91054, Germany.
Matthias VorgerdDepartment of Neurology, BG-University Hospital Bergmannsheil, Ruhr-University Bochum, 44789, Bochum, Germany.
Lara SchlaffkeDepartment of Neurology, BG-University Hospital Bergmannsheil, Ruhr-University Bochum, 44789, Bochum, Germany.
Joachim WoelfleDepartment of Pediatrics and Adolescent Medicine, University Hospital Erlangen, Friedrich-Alexander-Universität (FAU) Erlangen-Nürnberg, Erlangen, 91054, Germany.
Andreas HahnDepartment of Child Neurology, Justus-Liebig-Universität Giessen, 35385, Giessen, Germany.
Alexander MenschDepartment of Neurology, Martin-Luther-Universität Halle-Wittenberg, 06120, Halle (Saale), Germany.ORCID 0000-0003-0089-4473
Martin WinterhollerSana Krankenhaus Rummelsberg, 90489, Nuremberg/Schwarzenbruck, Germany.
Regina TrollmannDepartment of Pediatrics and Adolescent Medicine, University Hospital Erlangen, Friedrich-Alexander-Universität (FAU) Erlangen-Nürnberg, Erlangen, 91054, Germany.ORCID 0000-0001-6031-8537
Rafael HeißInstitute of Radiology, University Hospital Erlangen, Friedrich-Alexander-Universität (FAU) Erlangen-Nürnberg, Erlangen, 91054, Germany.ORCID 0000-0002-2897-5411
Alexandra L Wagner *Department of Pediatrics and Adolescent Medicine, University Hospital Erlangen, Friedrich-Alexander-Universität (FAU) Erlangen-Nürnberg, Erlangen, 91054, Germany.
Roman Raming *Department of Pediatrics and Adolescent Medicine, University Hospital Erlangen, Friedrich-Alexander-Universität (FAU) Erlangen-Nürnberg, Erlangen, 91054, Germany.
Ferdinand Knieling *Department of Pediatrics and Adolescent Medicine, University Hospital Erlangen, Friedrich-Alexander-Universität (FAU) Erlangen-Nürnberg, Erlangen, 91054, Germany. ferdinand.knieling@uk-erlangen.de.ORCID 0000-0002-3535-2626

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Pompe disease (PD) is a rare autosomal recessive glycogen storage disorder that causes proximal muscle weakness and loss of respiratory function. While enzyme replacement therapy (ERT) is the only effective treatment, biomarkers for disease monitoring are scarce. Following ex vivo biomarker validation in phantom studies, we apply multispectral optoacoustic tomography (MSOT), a laser- and ultrasound-based non-invasive imaging approach, in a clinical trial (NCT05083806) to image the biceps muscles of 10 late-onset PD (LOPD) patients and 10 matched healthy controls. MSOT is compared with muscle magnetic resonance imaging (MRI), ultrasound, spirometry, muscle testing and quality of life scores. Next, results are validated in an independent LOPD patient cohort from a second clinical site. Our study demonstrates that MSOT enables imaging of subcellular disease pathology with increases in glycogen/water, collagen and lipid signals, providing higher sensitivity in detecting muscle degeneration than current methods. This translational approach suggests implementation in the complex care of these rare disease patients.

Indexed as

GlycogenGlycogen Storage Disease Type IIMagnetic Resonance ImagingPhotoacoustic TechniquesAdultCase-Control StudiesFemaleHumansMaleMiddle AgedMuscle, SkeletalPhantoms, ImagingUltrasonographyGlycogen

Identifiers

PMID39245687
PMCPMC11381542

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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.