Evidence map›Paper›PMID 23677550›Full record

ArticleMuscle & nerve2013

The cooperative international neuromuscular research group Duchenne natural history study--a longitudinal investigation in the era of glucocorticoid therapy: design of protocol and the methods used.

Craig M McDonald, Erik K Henricson, R Ted Abresch, Jay J Han, Diana M Escolar, Julaine M Florence, Tina Duong, Adrienne Arrieta, Paula R Clemens, Eric P Hoffman and 2 more

3 registry-linked trialsAbstract readMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Article in Muscle & nerve, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 3 registered trials, which are not on this map. Cited by 101 papers, 4 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
101citing papers in PubMed, 4 pooled it
–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.

NCT03439670 phase2completednot on this mapstarted 2018, after this paper: background citation

A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study With Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)

TypeinterventionalSponsorReveraGen BioPharma, Inc.Ran2018 to 2021Enrolled121ConditionsDuchenne Muscular DystrophyArmsVamorolone, Prednisone, Placebo
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)
NCT06290713 phase2recruitingnot on this mapstarted 2024, after this paper: background citation

Vasodilators and Exercise as Adjuvant Therapy for Duchenne Muscular Dystrophy (VASO-REx Study)

TypeinterventionalSponsorUniversity of FloridaRan2024 to 2026Enrolled50ConditionsDuchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy, Muscular Dystrophy in ChildrenArmsTadalafil, Placebo, Exercise Training
3 · Its place in the literature

Who cites it

101 citing papers in PubMed, 4 syntheses or guidelines pooled it.

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41 more citing papers are in PubMed but not listed here.

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.

Craig M McDonaldDepartment of Physical Medicine & Rehabilitation, School of Medicine, University of California, Davis, 4860 Y Street, Suite 3850, Sacramento, California 95817, USA. cmmcdonald@ucdavis.edu
Erik K Henricson
R Ted Abresch
Jay J Han
Diana M Escolar
Julaine M Florence
Tina Duong
Adrienne Arrieta
Paula R Clemens
Eric P Hoffman
Avital Cnaan
Cinrg Investigators

Funding

Washington University Institute of Clinical and Translational Sciences (UL1)UL1RR024992 · NCRR · WASHINGTON UNIVERSITY · PI EVANOFF, BRADLEY A · 2007 to 2011
$45.4M
Washington University Institute of Clinical and Translational SciencesUL1TR000448 · NCATS · WASHINGTON UNIVERSITY · PI EVANOFF, BRADLEY A · 2012 to 2016
$41.4M
SUPPLEMENT FOR RENOVATION OF ANIMAL FACILITIES AT INSTITUTE OF NEUROBIOLOGYG12RR003051 · NCRR · UNIVERSITY OF PUERTO RICO MED SCIENCES · PI FERNANDEZ-REPOLLET, EMMA · 1986 to 2011
$28.1M
TRANSLATIONAL PROTEOMICS CENTERG12MD007600 · NIMHD · UNIVERSITY OF PUERTO RICO MED SCIENCES · PI FERNANDEZ-REPOLLET, EMMA · 2012 to 2016
$11.0M
TRCLU54RR026139 · NCRR · UNIVERSITY OF PUERTO RICO MED SCIENCES · PI FRONTERA, WALTER R. · 2010 to 2011
$8.4M
CTSA INFRASTRUCTURE FOR PEDIATRIC RESEARCHUL1RR031988 · NCRR · CHILDREN'S RESEARCH INSTITUTE · PI GUAY-WOODFORD, LISA MARIE · 2010 to 2011
$8.2M
Wellstone Muscular Dystrophy Center: Children's Nat'l Med CtrU54HD053177 · NICHD · CHILDREN'S RESEARCH INSTITUTE · PI HOFFMAN, ERIC P. · 2005 to 2009
$7.3M
Biomarker discovery and validation in a Duchenne dystrophy natural history studyR01AR062380 · NIAMS · UNIVERSITY OF CALIFORNIA AT DAVIS · PI HATHOUT, YETRIB, MCDONALD, CRAIG M. · 2011 to 2014
$1.5M
Clinically Meaningful Outcomes for Duchenne Muscular Dystrophy Therapeutic TrialsR01AR061875 · NIAMS · UNIVERSITY OF CALIFORNIA AT DAVIS · PI MCDONALD, CRAIG M. · 2011 to 2014
$1.3M
NCATS NIH HHS UL1 TR000448NCRR NIH HHS G12 RR003051NCRR NIH HHS G12RR003051NCRR NIH HHS U54 RR026139NCRR NIH HHS U54RR026139NCRR NIH HHS UL1 RR024992NCRR NIH HHS UL1RR024992NCRR NIH HHS UL1 RR031988NCRR NIH HHS UL1RR031988NIAMS NIH HHS 1R01AR061875NIAMS NIH HHS R01 AR061875NIAMS NIH HHS R01 AR062380NIAMS NIH HHS R01AR062380NICHD NIH HHS U54 HD053177NICHD NIH HHS U54HD053177NIMHD NIH HHS G12 MD007600
6 · The paper itself

Abstract

unlabelledContemporary natural history data in Duchenne muscular dystrophy (DMD) is needed to assess care recommendations and aid in planning future trials.

methodsThe Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study (DMD-NHS) enrolled 340 individuals, aged 2-28 years, with DMD in a longitudinal, observational study at 20 centers. Assessments obtained every 3 months for 1 year, at 18 months, and annually thereafter included: clinical history; anthropometrics; goniometry; manual muscle testing; quantitative muscle strength; timed function tests; pulmonary function; and patient-reported outcomes/health-related quality-of-life instruments.

resultsGlucocorticoid (GC) use at baseline was 62% present, 14% past, and 24% GC-naive. In those ≥6 years of age, 16% lost ambulation over the first 12 months (mean age 10.8 years).

conclusionsDetailed information on the study methodology of the CINRG DMD-NHS lays the groundwork for future analyses of prospective longitudinal natural history data. These data will assist investigators in designing clinical trials of novel therapeutics.

Indexed as

International CooperationResearch DesignAdolescentAdultChildChild, PreschoolGlucocorticoidsHumansInternationalityLongitudinal StudiesMaleMuscular Dystrophy, DuchenneProspective StudiesYoung AdultGlucocorticoids

Identifiers

PMID23677550
PMCPMC4147958

What OpenQuestion holds

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LicenceTDM
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.