Evidence map›Paper›PMID 41720458›Full record

ArticleTranslational research : the journal of laboratory and clinical medicine2026

Mapping glycogen accumulation and treatment effect in Pompe disease with saturation transfer MRI.

Qing Zeng, Yuguo Li, Derek Timm, Tyler Johnson, Nickita Mehta, Lukas Martin, Brian Fox, Peter C M van Zijl, Glenn A Walter, Jens T Rosenberg and 6 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Translational research : the journal of laboratory and clinical medicine, 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

16 authors.

Qing ZengRussell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins University School of Medicine, Baltimore, MD, United States; F.M. Kirby Research Center for Functional Brain Imaging, Kennedy Krieger Institute, Baltimore, MD, United States.
Yuguo LiRussell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins University School of Medicine, Baltimore, MD, United States; F.M. Kirby Research Center for Functional Brain Imaging, Kennedy Krieger Institute, Baltimore, MD, United States.
Derek TimmAmicus Therapeutics Inc., Princeton, NJ, United States.
Tyler JohnsonAmicus Therapeutics Inc., Princeton, NJ, United States.
Nickita MehtaAmicus Therapeutics Inc., Princeton, NJ, United States.
Lukas MartinAmicus Therapeutics Inc., Princeton, NJ, United States.
Brian FoxAmicus Therapeutics Inc., Princeton, NJ, United States.
Peter C M van ZijlRussell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins University School of Medicine, Baltimore, MD, United States; F.M. Kirby Research Center for Functional Brain Imaging, Kennedy Krieger Institute, Baltimore, MD, United States.
Glenn A WalterDepartment of Physiology and Functional Genomics, University of Florida, Gainesville, FL, United States.
Jens T RosenbergAdvanced Magnetic Resonance Imaging and Spectroscopy Facility, McKnight Brain Institute, University of Florida, Gainesville, FL, United States.
Craig W Vander KooiDepartment of Biochemistry and Molecular Biology, College of Medicine, University of Florida, Gainesville, FL, United States; Center for Advanced Spatial Biomolecule Research, University of Florida, Gainesville, FL, United States.
Manuela CortiDepartment of Pediatrics, University of Florida, Gainesville, FL, United States.
Matthew S GentryDepartment of Biochemistry and Molecular Biology, College of Medicine, University of Florida, Gainesville, FL, United States; Center for Advanced Spatial Biomolecule Research, University of Florida, Gainesville, FL, United States.
Ramon C SunDepartment of Biochemistry and Molecular Biology, College of Medicine, University of Florida, Gainesville, FL, United States; Center for Advanced Spatial Biomolecule Research, University of Florida, Gainesville, FL, United States.
Barry J ByrneDepartment of Pediatrics, University of Florida, Gainesville, FL, United States.
Nirbhay N YadavRussell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins University School of Medicine, Baltimore, MD, United States; F.M. Kirby Research Center for Functional Brain Imaging, Kennedy Krieger Institute, Baltimore, MD, United States. Electronic address: nyadav@jhu.edu.

Funding

TRD 4: Platforms for multi-modal and multi-scale imaging dataP41EB031771 · HUGO W. MOSER RES INST KENNEDY KRIEGER · 2025 to 2025
$1.2M
Non-Invasive Imaging of Neurological Glycogen Storage DiseaseR01NS127280 · HUGO W. MOSER RES INST KENNEDY KRIEGER · 2025 to 2025
$415k
NIBIB NIH HHS P41 EB031771NIH HHS S10 OD032188NINDS NIH HHS R01 NS127280
6 · The paper itself

Abstract

Pompe disease is a glycogen storage disease caused by the impaired breakdown of glycogen in lysosomes, leading to abnormal glycogen accumulation in tissue. Here we use glycogen nuclear Overhauser effect (glycoNOE) MRI to detect glycogen levels in skeletal muscle in a mouse model of Pompe disease. Moreover, we evaluated if glycoNOE MRI could detect changes in glycogen load after enzyme replacement therapy. The results show that glycoNOE MRI can distinguish between Pompe mice and wildtype controls. Furthermore, the technique detected treatment-dependent changes in muscle glycoNOE signals, which were validated with ex vivo biochemical assays. To demonstrate potential human translation, glycoNOE MRI was applied to two Pompe patients and revealed elevated glycogen levels in patients compared to healthy controls.

Indexed as

GlycogenGlycogen Storage Disease Type IIMagnetic Resonance ImagingAnimalsDisease Models, AnimalEnzyme Replacement TherapyFemaleHumansMaleMiceMuscle, SkeletalGlycogenGlycogenMusclePompe diseaseSaturation transfer MRI

Identifiers

PMID41720458
PMCPMC13001076

What Socratic holds

Textmetadata
LicenceTDM
Read underepoch 390

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 Socratic graph.