Evidence map›Paper›PMID 29398641›Full record

ReviewThe Lancet. Neurology2018

Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care, emergency management, psychosocial care, and transitions of care across the lifespan.

David J Birnkrant, Katharine Bushby, Carla M Bann, Susan D Apkon, Angela Blackwell, Mary K Colvin, Linda Cripe, Adrienne R Herron, Annie Kennedy, Kathi Kinnett and 8 more

2 registry-linked trialsOpen access · greenAbstract 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. It is linked to 2 registered trials, which are not on this map. Cited by 216 papers, 6 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
216citing papers in PubMed, 6 pooled it
16.5field-weighted citation impact, top 1% 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.

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
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

216 citing papers in PubMed, 6 syntheses or guidelines pooled it, 435 citations in OpenAlex.

  1. Pooled it
  2. Neurology · 2023
    Pooled it
  3. Pooled it
  4. Pooled it
  5. Pooled it
  6. Guideline
  7. Trial
  8. Trial
  9. Trial
  10. Trial
  11. Trial
  12. Early phase 2 trial of TAS-205 in patients with Duchenne muscular dystrophy.Annals of clinical and translational neurology · 2020
    Trial
  13. Review
  14. Article
  15. Article
  16. Review
  17. Article
  18. Article
  19. Article
  20. Review

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

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

18 authors at 15 institutions in 3 countries.

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.
Mary K ColvinMassachusetts General Hospital and Harvard Medical School, Boston, MA, USA.
Linda CripeDepartment of Pediatrics, Nationwide Children's Hospital, The Ohio State University, Columbus, OH, USA.
Adrienne R HerronRare Disorders and Health Outcomes Team, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GA, USA; Oak Ridge Institute for Science and Education, Oak Ridge, TN, USA.
Annie KennedyParent Project Muscular Dystrophy, Hackensack, NJ, USA.
Kathi KinnettParent Project Muscular Dystrophy, Hackensack, NJ, USA.
James NaprawaEmergency Department, University of California San Francisco Benioff Children's Hospital, Oakland, CA, USA.
Garey NoritzDepartment of Pediatrics, Nationwide Children's Hospital, The Ohio State University, Columbus, OH, USA.
James PoyskyBaylor College of Medicine, Houston, TX, 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.
Christina J TroutStead Family Department of Pediatrics, University of Iowa, Iowa City, IA, USA.
David R WeberDivision of Endocrinology and Diabetes, Golisano Children's Hospital, University of Rochester Medical Center, Rochester, NY, USA.
Leanne M WardDivision of Endocrinology and Metabolism, Children's Hospital of Eastern Ontario, and University of Ottawa, Ottawa, ON, Canada.
DMD Care Considerations Working Group
Parent Project Muscular Dystrophy · USRTI International · USBaylor College of Medicine · USCenters for Disease Control and Prevention · USGolisano Children's Hospital · USHarvard University · USMetroHealth Medical Center · USNational Center on Birth Defects and Developmental Disabilities · USNationwide Children's Hospital · USNewcastle University · GBSeattle Children's Hospital · USThe Ohio State University · USUniversity of California, San Francisco · USUniversity of Iowa · USUniversity of Ottawa · CA

Funding

Intramural CDC HHS CC999999
6 · The paper itself

Abstract

Improvements in the function, quality of life, and longevity of patients with Duchenne muscular dystrophy (DMD) have been achieved through a multidisciplinary approach to management across a range of health-care specialties. In part 3 of this update of the DMD care considerations, we focus on primary care, emergency management, psychosocial care, and transitions of care across the lifespan. Many primary care and emergency medicine clinicians are inexperienced at managing the complications of DMD. We provide a guide to the acute and chronic medical conditions that these first-line providers are likely to encounter. With prolonged survival, individuals with DMD face a unique set of challenges related to psychosocial issues and transitions of care. We discuss assessments and interventions that are designed to improve mental health and independence, functionality, and quality of life in critical domains of living, including health care, education, employment, interpersonal relationships, and intimacy.

Indexed as

Continuity of Patient CareMental Health ServicesPractice Guidelines as TopicQuality of LifeBehavioral SymptomsEmergency Medical ServicesHumansMuscular Dystrophy, DuchennePrimary Health Care

Identifiers

PMID29398641
PMCPMC5902408
OpenAlexW2784397775

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.