Evidence map›Paper›PMID 30198876›Full record

ArticleJournal of neuromuscular diseases2018

"Of Mice and Measures": A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic.

Heather Gordish-Dressman, Raffaella Willmann, Laura Dalle Pazze, Arati Kreibich, Maaike van Putten, Ahlke Heydemann, Laurent Bogdanik, Cathleen Lutz, Kay Davies, Alexis R Demonbreun and 20 more

Abstract readConference Proceedings
In one paragraph

Article in Journal of neuromuscular diseases, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers.

0numbers the graph read from it
0cells of the map it votes in
28citing 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

28 citing papers in PubMed.

  1. Review
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  4. The D2.B10-DmdThe American journal of pathology · 2026
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  19. Mouse Models of Muscle Fibrosis.Methods in molecular biology (Clifton, N.J.) · 2021
    Article
  20. Review
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

30 authors.

Heather Gordish-DressmanChildren's National Medical Center, Washington, DC, USA.
Raffaella WillmannBiozentrum, University of Basel, Basel, Switzerland.
Laura Dalle PazzeCharley's Fund, New York, NY, USA.
Arati KreibichCharley's Fund, New York, NY, USA.
Maaike van PuttenDepartment of Human Genetics, Leiden University Medical Center, the Netherlands.
Ahlke HeydemannDepartment of Physiology and Biophysics, University of Illinois at Chicago, Chicago, IL, USA.
Laurent BogdanikThe Jackson Laboratory, Bar Harbor, ME, USA.
Cathleen LutzThe Jackson Laboratory, Bar Harbor, ME, USA.
Kay DaviesDepartment of Physiology, University of Oxford, Anatomy and Genetics, Oxford, UK.
Alexis R DemonbreunCenter for Genetic Medicine, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.
Dongsheng DuanDepartment of Molecular Microbiology and Immunology, Department of Neurology, Department of Biomedical Sciences and Department of Bioengineering, University of Missouri, Columbia, MO, USA.
David ElseySummit Therapeutics, Abingdon, Oxfordshire, UK.
So-Ichiro FukadaLaboratory of Molecular and Cellular Physiology, Graduate School of Pharmaceutical Sciences, Osaka University, Osaka, Japan.
Mahasweta GirgenrathRare Disease Research Unit, Pfizer, CA, USA.
J Patrick GonzalezSolid Biosciences, Cambridge, MA, USA.
Miranda D GroundsSchool of Human Science, the University of Western Australia, Perth, Australia.
Andy NicholsCatabasis Pharmaceuticals, Cambridge, MA, USA.
Terry PartridgeChildren's National Medical Center, Washington, DC, USA.
Marco PassiniSarepta Therapeutics, Inc. Cambridge, MA, USA.
Francesca SanaricaDepartment of Pharmacy and Drug Sciences, Unit of Pharmacology, University of Bari "Aldo Moro", Italy.
Frederick J SchnellSarepta Therapeutics, Inc. Cambridge, MA, USA.
Dominic J WellsDepartment of Comparative Biomedical Sciences, Royal Veterinary College, London, UK.
Toshifumi YokotaDepartment of Medical Genetics, University of Alberta, Canada.
Courtney S YoungDepartment of Neurology, Molecular Biology Institute, Center for Duchenne Muscular Dystrophy at UCLA, University of California, Los Angeles, CA, USA.
Zhong ZhongWave Life Sciences, Cambridge, MA, USA.
Christopher SpurneyChildren's National Medical Center, Washington, DC, USA.
Melissa SpencerDepartment of Neurology, Molecular Biology Institute, Center for Duchenne Muscular Dystrophy at UCLA, University of California, Los Angeles, CA, USA.
Annamaria De LucaDepartment of Pharmacy and Drug Sciences, Unit of Pharmacology, University of Bari "Aldo Moro", Italy.
Kanneboyina NagarajuSchool of Pharmacy and Pharmaceutical Sciences, Binghamton University, New York, USA.
Annemieke Aartsma-RusDepartment of Human Genetics, Leiden University Medical Center, the Netherlands.

Funding

Whole body single AAV microgene therapy in canine DMDR01NS090634 · NINDS · UNIVERSITY OF MISSOURI-COLUMBIA · PI Dongsheng Duan · 2015 to 2026
$4.2M
CRISPR/Cas9-Based Gene Editing for the Correction of Duchenne Muscular DystrophyR01AR069085 · NIAMS · DUKE UNIVERSITY · PI GERSBACH, CHARLES A. · 2016 to 2020
$1.7M
Preclinical drug trial in mouse models of inflammationK26OD011171 · OD · STATE UNIVERSITY OF NY,BINGHAMTON · PI NAGARAJU, KANNEBOYINA · 2012 to 2015
$420k
R16/17-Independent nNOS Anchoring MechanismR21AR067985 · NIAMS · UNIVERSITY OF MISSOURI-COLUMBIA · PI DUAN, DONGSHENG · 2016 to 2017
$355k
Medical Research Council MR/N010698/1NIAMS NIH HHS R01 AR069085NIAMS NIH HHS R21 AR067985NIH HHS K26 OD011171NINDS NIH HHS R01 NS090634
6 · The paper itself

Abstract

A new line of dystrophic mdx mice on the DBA/2J (D2) background has emerged as a candidate to study the efficacy of therapeutic approaches for Duchenne muscular dystrophy (DMD). These mice harbor genetic polymorphisms that appear to increase the severity of the dystropathology, with disease modifiers that also occur in DMD patients, making them attractive for efficacy studies and drug development. This workshop aimed at collecting and consolidating available data on the pathological features and the natural history of these new D2/mdx mice, for comparison with classic mdx mice and controls, and to identify gaps in information and their potential value. The overall aim is to establish guidance on how to best use the D2/mdx mouse model in preclinical studies.

Indexed as

Disease Models, AnimalMuscular Dystrophy, AnimalMuscular Dystrophy, DuchenneAnimalsMiceMice, Inbred DBAMice, Inbred mdxDBA/2JDMDDuchenne muscular dystrophymdx

Identifiers

PMID30198876
PMCPMC6218134

What OpenQuestion holds

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Registered trials

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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.