Evidence map›Paper›PMID 33762424›Full record

ReviewOpen heart2021

Current state of cardiac troponin testing in Duchenne muscular dystrophy cardiomyopathy: review and recommendations from the Parent Project Muscular Dystrophy expert panel.

Christopher F Spurney, Deborah Ascheim, Lawrence Charnas, Linda Cripe, Kan Hor, Nicholas King, Kathi Kinnett, Elizabeth M McNally, John-Michael Sauer, Lee Sweeney and 2 more

Registry-linked trialOpen access · goldAbstract readReview
In one paragraph

Review in Open heart, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05753462 (Phase 1/2a, Monocentric, Open Label Study to Evaluate the Safety, PK and PD of SQY51 in Paediatric and Adult Patients With a Genetically Confirmed Diagnosis of Duchenne Muscular Dystrophy), which is not on this map. Cited by 16 papers.

0numbers the graph read from it
0cells of the map it votes in
16citing papers in PubMed
1.7field-weighted citation impact, top 15% of its field
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.

NCT05753462 phase1 / phase2unknown statusnot on this mapstarted 2023, after this paper: background citation

Phase 1/2a, Monocentric, Open Label Study to Evaluate the Safety, PK and PD of SQY51 in Paediatric and Adult Patients With a Genetically Confirmed Diagnosis of Duchenne Muscular Dystrophy

TypeinterventionalSponsorSqy TherapeuticsRan2023 to 2025Enrolled12ConditionsDuchenne Muscular DystrophyArmsPhase 1, SQY51, Phase 2a, SQY51 (cohort 1), Phase 2a, SQY51 (cohort 2), Phase 2a, SQY51 (cohort 3)
3 · Its place in the literature

Who cites it

16 citing papers in PubMed, 24 citations in OpenAlex.

  1. Serial assessment of echocardiographic measures in Friedreich ataxia.American heart journal plus : cardiology research and practice · 2026
    Article
  2. Article
  3. Review
  4. Article
  5. Review
  6. Article
  7. Review
  8. Article
  9. Article
  10. Review
  11. Article
  12. Article
  13. Mechanisms of reduced myocardial energetics of the dystrophic heart.American journal of physiology. Heart and circulatory physiology · 2024
    Article
  14. Article
  15. Review
  16. Characterization of the Cardiac Structure and Function of Conscious D2.B10-International journal of molecular sciences · 2023
    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

12 authors at 9 institutions in 4 countries.

Christopher F SpurneyChildren's National Heart Institute, Children's National Hospital, Washington, District of Columbia, USA cspurney@childrensnational.org.ORCID 0000-0003-2493-5735
Deborah AscheimD2A, Los Angeles, California, USA.
Lawrence CharnasClinical Research Rare Neurology Disease, Pfizer, Cambridge, Massachusetts, USA.ORCID 0000-0002-4133-7959
Linda CripeThe Heart Center, Nationwide Children's Hospital, Columbus, Ohio, USA.
Kan HorThe Heart Center, Nationwide Children's Hospital, Columbus, Ohio, USA.
Nicholas KingCritical Path Institute, Tucson, Arizona, USA.
Kathi KinnettParent Project Muscular Dystrophy, Hackensack, New Jersey, USA.
Elizabeth M McNallyCenter for Genetic Medicine, Northwestern Medicine, Chicago, Illinois, USA.
John-Michael SauerCritical Path Institute, Tucson, Arizona, USA.
Lee SweeneyDepartment of Pharmacology and Therapeutics, University of Florida, Gainesville, Florida, USA.
Chet VillaCardiology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
Larry W MarkhamPediatrics, Division of Pediatric Cardiology, Riley Hospital for Children at Indiana University Health, Indianapolis, Indiana, USA.
Critical Path Institute · USNationwide Children's Hospital · USCenter for Human Genetics · USChildren's National · USCincinnati Children's Hospital Medical Center · USIndiana University Health · USParent Project Muscular Dystrophy · USPfizer (United States) · USUniversity of Florida · US

Funding

Northwestern University Clinical and Translational Science Institute (NUCATS)UL1TR001422 · NCATS · NORTHWESTERN UNIVERSITY AT CHICAGO · PI D'AQUILA, RICHARD · 2015 to 2023
$56.8M
NCATS NIH HHS UL1 TR001422
6 · The paper itself

Abstract

Cardiac disease is now the leading cause of death in Duchenne muscular dystrophy (DMD). Clinical evaluations over time have demonstrated asymptomatic cardiac troponin elevations and acute elevations are associated with symptoms and cardiac dysfunction in DMD. Clinicians require a better understanding of the relationship of symptoms, troponin levels and progression of cardiac disease in DMD. As clinical trials begin to assess novel cardiac therapeutics in DMD, troponin levels in DMD are important for safety monitoring and outcome measures. The Parent Project Muscular Dystrophy convened an expert panel of cardiologists, scientists, and regulatory and industry specialists on 16 December 2019 in Silver Spring, Maryland and reviewed published and unpublished data from their institutions. The panel recommended retrospective troponin data analyses, prospective longitudinal troponin collection using high-sensitivity cardiac troponin I assays, inclusion of troponin in future clinical trial outcomes and future development of clinical guidelines for monitoring and treating troponin elevations in DMD.

Indexed as

ParentsPractice Guidelines as TopicBiomarkersCardiomyopathiesDisease ProgressionEchocardiographyHumansMuscular Dystrophy, DuchenneTroponin IBiomarkersTroponin Ibiomarkerscardiomyopathydilatedgenetic diseasesinborn

Identifiers

PMID33762424
PMCPMC7993361
OpenAlexW3138453819

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

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.