Evidence map›Paper›PMID 39567576›Full record

ArticleScientific reports2024

Serum protein and imaging biomarkers after intermittent steroid treatment in muscular dystrophy.

Alexander B Willis, Aaron S Zelikovich, Robert Sufit, Senda Ajroud-Driss, Krista Vandenborne, Alexis R Demonbreun, Abhinandan Batra, Glenn A Walter, Elizabeth M McNally

Abstract read
In one paragraph

Article in Scientific reports, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing 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

7 citing papers in PubMed.

  1. Review
  2. Article
  3. Article
  4. Observational
  5. Review
  6. Serum protein biomarker signature of Duchenne muscular dystrophy.European journal of translational myology · 2025
    Article
  7. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

9 authors.

Alexander B WillisCenter for Genetic Medicine, Northwestern University Feinberg School of Medicine, 303 E Superior SQ 5-516, Chicago, IL, 60611, USA.
Aaron S ZelikovichCenter for Genetic Medicine, Northwestern University Feinberg School of Medicine, 303 E Superior SQ 5-516, Chicago, IL, 60611, USA.
Robert SufitDepartment of Neurology, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.
Senda Ajroud-DrissDepartment of Neurology, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.
Krista VandenborneDepartment of Physical Therapy, University of Florida, Gainesville, FL, USA.
Alexis R DemonbreunCenter for Genetic Medicine, Northwestern University Feinberg School of Medicine, 303 E Superior SQ 5-516, Chicago, IL, 60611, USA.
Abhinandan BatraDepartment of Physical Therapy, University of Louisiana at Monroe, Monroe, LA, USA.
Glenn A WalterDepartment of Physiology and Aging, University of Florida, Gainesville, FL, USA.
Elizabeth M McNallyCenter for Genetic Medicine, Northwestern University Feinberg School of Medicine, 303 E Superior SQ 5-516, Chicago, IL, 60611, USA. elizabeth.mcnally@northwestern.edu.

Funding

Training and education CoreU54AR052646 · NIAMS · UNIVERSITY OF PENNSYLVANIA · PI MCNALLY, ELIZABETH M, SWEENEY, H LEE · 2005 to 2020
$24.1M
SARCOGLYCAN IN MYOPATHY AND MUSCLE MEMBRANE STABILITYR01HL061322 · NHLBI · UNIVERSITY OF CHICAGO · PI MCNALLY, ELIZABETH M · 1999 to 2022
$7.5M
Myoferlin in Muscle Membrane Fusion and RepairR01NS047726 · NINDS · UNIVERSITY OF CHICAGO · PI ALEXIS R. DEMONBREUN, Elizabeth M McNally · 2003 to 2026
$7.2M
Understanding and Improving Therapies for the Muscular Dystrophies through Noninvasive BiomarkersP50AR052646 · NIAMS · UNIVERSITY OF FLORIDA · PI SWEENEY, H LEE · 2021 to 2024
$6.2M
Development of anti-LTBP4 as a biologic to treat Neuromuscular DiseasesUG3NS127383 · NINDS · NORTHWESTERN UNIVERSITY AT CHICAGO · PI DEMONBREUN, ALEXIS R., MCNALLY, ELIZABETH M · 2022 to 2023
$2.1M
Development of anti-LTBP4 as a biologic to treat Neuromuscular DiseasesUH3NS127383 · NINDS · NORTHWESTERN UNIVERSITY AT CHICAGO · PI DEMONBREUN, ALEXIS R., MCNALLY, ELIZABETH M · 2024 to 2024
$616k
NHLBI NIH HHS R01 HL061322NIAMS NIH HHS P50 AR052646NIAMS NIH HHS U54 AR052646NINDS NIH HHS R01 NS047726NINDS NIH HHS UG3 NS127383NINDS NIH HHS UH3 NS127383
6 · The paper itself

Abstract

Weekly Steroids in Muscular Dystrophy (WSiMD) was a pilot study to evaluate once weekly prednisone in patients with Limb Girdle and Becker muscular dystrophy (LGMD and BMD, respectively). At study endpoint, there were trends towards increased lean mass, reduced fat mass, reduced creatine kinase and improved motor function. The investigation was motivated by studies in mouse muscular dystrophy models in which once weekly glucocorticoid exposure enhanced muscle strength and reduced fibrosis. WSiMD participants provided blood samples for aptamer serum profiling at baseline and after 6 months of weekly steroids. A subset completed magnetic resonance (MR) evaluation of muscle at study onset and endpoint. At baseline compared to age and sex-matched healthy controls, the aggregate serum protein profile in the WSiMD cohort was dominated by muscle proteins, reflecting leak of muscle proteins into serum. Disease status produced more proteins differentially present in serum compared to steroid-treatment effect. Nonetheless, a response to prednisone was discernable in the WSiMD cohort, even at this low dose. Glucocorticoids decreased muscle proteins and increased certain immune process- and matrix-associated proteins. Muscle MR fat fraction showed trends with functional status. The prednisone-responsive markers could be used in larger trial of prednisone efficacy.

Indexed as

BiomarkersMuscular Dystrophy, DuchennePrednisoneAdultBlood ProteinsFemaleGlucocorticoidsHumansMagnetic Resonance ImagingMaleMiddle AgedMuscle, SkeletalMuscular Dystrophies, Limb-GirdlePilot ProjectsYoung AdultBiomarkersBlood ProteinsGlucocorticoidsPrednisoneGlucocorticoidLimb girdle muscular dystrophyMRIMuscleMuscular dystrophyPrednisoneSerum biomarkers

Identifiers

PMID39567576
PMCPMC11579281

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
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.