Evidence map›Paper›PMID 23137739›Full record

ReviewPhysical medicine and rehabilitation clinics of North America2012

Novel approaches to corticosteroid treatment in Duchenne muscular dystrophy.

Eric P Hoffman, Erica Reeves, Jesse Damsker, Kanneboyina Nagaraju, John M McCall, Edward M Connor, Kate Bushby

Open access · greenAbstract readReview
In one paragraph

Review in Physical medicine and rehabilitation clinics of North America, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 32 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
32citing papers in PubMed, 2 pooled it
2.6field-weighted citation impact, top 10% 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.

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

32 citing papers in PubMed, 2 syntheses or guidelines pooled it, 65 citations in OpenAlex.

  1. Pooled it
  2. Guideline
  3. Trial
  4. Article
  5. Article
  6. Review
  7. Article
  8. Article
  9. Review
  10. Review
  11. Science advances · 2021
    Article
  12. Disruption of a key ligand-H-bond network drives dissociative properties in vamorolone for Duchenne muscular dystrophy treatment.Proceedings of the National Academy of Sciences of the United States of America · 2020
    Article
  13. Review
  14. Review
  15. Article
  16. Article
  17. Article
  18. Article
  19. Recent developments in Duchenne muscular dystrophy: facts and numbers.Journal of cachexia, sarcopenia and muscle · 2017
    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

7 authors at 3 institutions in 2 countries.

Eric P HoffmanCenter for Genetic Medicine Research, Children's National Medical Center, Washington, DC 20010, USA. ehoffman@cnmcresearch.org
Erica Reeves
Jesse Damsker
Kanneboyina Nagaraju
John M McCall
Edward M Connor
Kate Bushby
Children's National · USReveraGen BioPharma (United States) · USCentre for Life · GB

Funding

FOR-DMD:Double-blind randomized trial to optimize steroid regimen in Duchenne MDU01NS061799 · NINDS · UNIVERSITY OF ROCHESTER · PI GRIGGS, ROBERT C, GUGLIERI, MICHELA · 2010 to 2019
$13.7M
Wellstone Muscular Dystrophy Center: Children's Nat'l Med CtrU54HD053177 · NICHD · CHILDREN'S RESEARCH INSTITUTE · PI HOFFMAN, ERIC P. · 2005 to 2009
$7.3M
Improved Diagnostic of the Muscular DystrophiesR01NS029525 · NINDS · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI HOFFMAN, ERIC P. · 1991 to 2015
$6.5M
NICHD NIH HHS U54 HD053177NICHD NIH HHS U54HD053177NINDS NIH HHS R01 NS029525NINDS NIH HHS U01 NS061799NINDS NIH HHS U01NS061799
6 · The paper itself

Abstract

Although prednisone has never been formally approved for use in Duchenne muscular dystrophy (DMD) by regulatory agencies, its efficacy has been confirmed in trials dating from the 1980s. There is a strong need for optimization of both specific type of glucocorticoid (eg, prednisone, vs deflazacort or others) and the dosing regimen. Ideally an optimized regimen would maximize efficacy while minimizing side-effect profiles. A new trial, FOR-DMD, aims to address this gap in knowledge. In parallel, there has been progress in the area of "dissociative steroids," drugs that are able to better separate efficacy and side effects, providing a broader therapeutic window.

Indexed as

Drug DiscoveryGlucocorticoidsHumansMuscular Dystrophy, DuchennePrednisonePregnenedionesdeflazacortGlucocorticoidsPrednisonePregnenediones

Identifiers

PMID23137739
PMCPMC3606917
OpenAlexW2013623387

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

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.