Evidence map›Paper›PMID 21078917›Full record

GuidelineJournal of child neurology2010

Consensus statement on standard of care for congenital muscular dystrophies.

Ching H Wang, Carsten G Bonnemann, Anne Rutkowski, Thomas Sejersen, Jonathan Bellini, Vanessa Battista, Julaine M Florence, Ulrike Schara, Pamela M Schuler, Karim Wahbi and 31 more

Registry-linked trialOpen access · greenAbstract readConsensus StatementPractice GuidelineReview
In one paragraph

Guideline in Journal of child neurology, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01403402 (Congenital Muscle Disease Patient and Proxy Reported Outcome Study), which is not on this map. Cited by 71 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
71citing papers in PubMed, 3 pooled it
5.8field-weighted citation impact, top 3% 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.

NCT01403402 recruitingnot on this map

Congenital Muscle Disease Patient and Proxy Reported Outcome Study

Typeobservational_patient_registrySponsorCure CMDRan2009 to 2029Enrolled4,000ConditionsCongenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency, Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Fatty Liver and Infantile-onset Cataract Caused by TRAPPC11 Mutations), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan)
3 · Its place in the literature

Who cites it

71 citing papers in PubMed, 3 syntheses or guidelines pooled it, 212 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Guideline
  4. Trial
  5. Trial
  6. Trial
  7. Article
  8. Article
  9. Article
  10. Article
  11. Article
  12. Observational
  13. Article
  14. Article
  15. The congenital muscular dystrophies.Annals of the Child Neurology Society · 2024
    Review
  16. Article
  17. Article
  18. Article
  19. Neurology. Genetics · 2023
    Article
  20. Review

11 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

41 authors at 20 institutions in 8 countries.

Ching H WangStanford University School of Medicine, Stanford, California, USA. wangch@stanford.edu
Carsten G Bonnemann
Anne Rutkowski
Thomas Sejersen
Jonathan Bellini
Vanessa Battista
Julaine M Florence
Ulrike Schara
Pamela M Schuler
Karim Wahbi
Annie Aloysius
Robert O Bash
Christophe Béroud
Enrico Bertini
Kate Bushby
Ronald D Cohn
Anne M Connolly
Nicolas Deconinck
Isabelle Desguerre
Michelle Eagle
Brigitte Estournet-Mathiaud
Ana Ferreiro
Albert Fujak
Nathalie Goemans
Susan T Iannaccone
Patricia Jouinot
Marion Main
Paola Melacini
Wolfgang Mueller-Felber
Francesco Muntoni
Leslie L Nelson
Jes Rahbek
Susana Quijano-Roy
Caroline Sewry
Kari Storhaug
Anita Simonds
Brian Tseng
Jiri Vajsar
Andrea Vianello
Reinhard Zeller
International Standard of Care Committee for Congenital Muscular Dystrophy
Hôpital Raymond-Poincaré · FRThe University of Texas Southwestern Medical Center · USKU Leuven · BENewcastle University · GBStanford University · USUniversity of Padua · ITBambino Gesù Children's Hospital · ITBoston College · USChildren's Hospital of Philadelphia · USCure CMD · USFriedrich-Alexander-Universität Erlangen-Nürnberg · DEHammersmith Hospital · GBHôpital Saint-Vincent-de-Paul · FRHospital for Sick Children · CAInserm · FRInstitut de Myologie · FRJohns Hopkins University · USKarolinska Institutet · SELudwig-Maximilians-Universität München · DEMassachusetts General Hospital · US

Funding

Molecular and Clinical Manifestations of Matrix and Aggregate MyopathiesZIANS003129 · NINDS · NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE · PI BÖNNEMANN, CARSTEN · 2011 to 2025
$52.8M
Intramural NIH HHS Z99 NS999999Intramural NIH HHS ZIA NS003129Medical Research Council G0601943Telethon GUP06004
6 · The paper itself

Abstract

Congenital muscular dystrophies are a group of rare neuromuscular disorders with a wide spectrum of clinical phenotypes. Recent advances in understanding the molecular pathogenesis of congenital muscular dystrophy have enabled better diagnosis. However, medical care for patients with congenital muscular dystrophy remains very diverse. Advances in many areas of medical technology have not been adopted in clinical practice. The International Standard of Care Committee for Congenital Muscular Dystrophy was established to identify current care issues, review literature for evidence-based practice, and achieve consensus on care recommendations in 7 areas: diagnosis, neurology, pulmonology, orthopedics/rehabilitation, gastroenterology/ nutrition/speech/oral care, cardiology, and palliative care. To achieve consensus on the care recommendations, 2 separate online surveys were conducted to poll opinions from experts in the field and from congenital muscular dystrophy families. The final consensus was achieved in a 3-day workshop conducted in Brussels, Belgium, in November 2009. This consensus statement describes the care recommendations from this committee.

Indexed as

Global HealthInternational CooperationChildChild, PreschoolClinical ProtocolsCongresses as TopicFemaleHumansMaleMuscular DystrophiesStandard of Care

Identifiers

PMID21078917
PMCPMC5207780
OpenAlexW2164867418

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.