Evidence map›Paper›PMID 29395989›Full record

ReviewThe Lancet. Neurology2018

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management.

David J Birnkrant, Katharine Bushby, Carla M Bann, Susan D Apkon, Angela Blackwell, David Brumbaugh, Laura E Case, Paula R Clemens, Stasia Hadjiyannakis, Shree Pandya and 6 more

Erratum issued 6 registry-linked trialsAbstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in The Lancet. Neurology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. It is linked to 6 registered trials, which are not on this map. Cited by 644 papers, 10 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
644citing papers in PubMed, 10 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.

NCT03680365 completednot on this map

Your Voice; Impact of DMD. A Qualitative Assessment of the Impact of DMD on the Lives of Families

TypeobservationalSponsorJett Foundation, Inc.Ran2018 to 2019Enrolled60ConditionsDuchenne Muscular Dystrophy, Burden, Dependency, Disability Physical, Disease Management
NCT03947112 completednot on this mapstarted 2020, after this paper: background citation

Physical Activity Level of Norwegian Boys with Duchenne Muscular Dystrophy - a Cross Sectional Study

TypeobservationalSponsorHaukeland University HospitalRan2020 to 2021Enrolled28ConditionsDuchenne Muscular DystrophyArmsPhysical activity registration
NCT04054375 phase2completednot on this mapstarted 2019, after this paper: background citation

Open Label Safety and Efficacy of Once Weekly Steroid in Patients With LGMD and Becker Muscular Dystrophy

TypeinterventionalSponsorNorthwestern UniversityRan2019 to 2022Enrolled20ConditionsLimb-girdle Muscular Dystrophy, Becker Muscular DystrophyArmsPrednisone
NCT05029232 naunknown statusnot on this mapstarted 2021, after this paper: background citation

Comprehensive Study of Duchenne Muscular Dystrophy at Sohag University Hospital

TypeinterventionalSponsorSohag UniversityRan2021 to 2023Enrolled50ConditionsDuchenne Muscular DystrophyArmsMLPA for duchenne
NCT05516745 naunknown statusnot on this mapstarted 2021, after this paper: background citation

E-monitoring of Pulmonary Function in Patients With Duchenne Muscular Dystrophy Undergoing Respiratory Rehabilitation at Home"

TypeinterventionalSponsorMedical University of GdanskRan2021 to 2025Enrolled200ConditionsDuchenne Muscular Dystrophy (DMD)ArmsAioCare spirometer, telerehabiliation of the respiratory system
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

644 citing papers in PubMed, 10 syntheses or guidelines pooled it.

  1. Pooled it
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  9. Meta-analysis of the efficacy and safety of vamorolone in Duchenne muscular dystrophy.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2025
    Pooled it
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  20. Review

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

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

16 authors.

David J BirnkrantDepartment of Pediatrics, MetroHealth Medical Center, Case Western Reserve University, Cleveland, OH, USA. Electronic address: dbirnkrant@metrohealth.org.
Katharine BushbyJohn Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK.
Carla M BannRTI International, Research Triangle Park, NC, USA.
Susan D ApkonDepartment of Rehabilitation Medicine, Seattle Children's Hospital, Seattle, WA, USA.
Angela BlackwellRTI International, Research Triangle Park, NC, USA.
David BrumbaughSection of Pediatric Gastroenterology, Hepatology, and Nutrition, Children's Hospital Colorado, Aurora, CO, USA.
Laura E CaseDoctor of Physical Therapy Division, Department of Orthopaedics, Duke University School of Medicine, Durham, NC, USA.
Paula R ClemensDepartment of Neurology, University of Pittsburgh School of Medicine, and Neurology Service, Department of Veterans Affairs Medical Center, Pittsburgh, PA, USA.
Stasia HadjiyannakisDivision of Endocrinology and Metabolism, Children's Hospital of Eastern Ontario, and University of Ottawa, Ottawa, ON, Canada.
Shree PandyaSchool of Medicine and Dentistry, University of Rochester, Rochester, NY, USA.
Natalie StreetRare Disorders and Health Outcomes Team, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GA, USA.
Jean TomezskoMedical Nutrition Consulting of Media LLC, and Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Kathryn R WagnerCenter for Genetic Muscle Disorders, Kennedy Krieger Institute, and Johns Hopkins School of Medicine, Baltimore, MD, USA.
Leanne M WardDivision of Endocrinology and Metabolism, Children's Hospital of Eastern Ontario, and University of Ottawa, Ottawa, ON, Canada.
David R WeberDivision of Endocrinology and Diabetes, Golisano Children's Hospital, University of Rochester Medical Center, Rochester, NY, USA.
DMD Care Considerations Working Group

Funding

Intramural CDC HHS CC999999
6 · The paper itself

Abstract

Since the publication of the Duchenne muscular dystrophy (DMD) care considerations in 2010, multidisciplinary care of this severe, progressive neuromuscular disease has evolved. In conjunction with improved patient survival, a shift to more anticipatory diagnostic and therapeutic strategies has occurred, with a renewed focus on patient quality of life. In 2014, a steering committee of experts from a wide range of disciplines was established to update the 2010 DMD care considerations, with the goal of improving patient care. The new care considerations aim to address the needs of patients with prolonged survival, to provide guidance on advances in assessments and interventions, and to consider the implications of emerging genetic and molecular therapies for DMD. The committee identified 11 topics to be included in the update, eight of which were addressed in the original care considerations. The three new topics are primary care and emergency management, endocrine management, and transitions of care across the lifespan. In part 1 of this three-part update, we present care considerations for diagnosis of DMD and neuromuscular, rehabilitation, endocrine (growth, puberty, and adrenal insufficiency), and gastrointestinal (including nutrition and dysphagia) management.

Indexed as

Disease ManagementMuscular Dystrophy, DuchenneEndocrine SystemGastrointestinal TractHumansNeuromuscular JunctionNutrition Therapy

Identifiers

PMID29395989
PMCPMC5869704

What OpenQuestion holds

Textmetadata
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.