Evidence mapPaperPMID 37534133Full record

ArticleiScience2023

Vamorolone improves Becker muscular dystrophy and increases dystrophin protein in

Nikki M McCormack, Nhu Y Nguyen, Christopher B Tully, Trinitee Oliver, Alyson A Fiorillo, Christopher R Heier

Open access · goldAbstract read
In one paragraph

Article in iScience, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
2.0field-weighted citation impact, top 14% 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

8 citing papers in PubMed, 13 citations in OpenAlex.

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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

6 authors at 1 institution in 1 country.

Nikki M McCormackCenter for Genetic Medicine Research, Children's National Hospital, Washington, DC, USA.
Nhu Y NguyenCenter for Genetic Medicine Research, Children's National Hospital, Washington, DC, USA.
Christopher B TullyCenter for Genetic Medicine Research, Children's National Hospital, Washington, DC, USA.
Trinitee OliverCenter for Genetic Medicine Research, Children's National Hospital, Washington, DC, USA.
Alyson A FiorilloCenter for Genetic Medicine Research, Children's National Hospital, Washington, DC, USA.
Christopher R HeierCenter for Genetic Medicine Research, Children's National Hospital, Washington, DC, USA.
Children's National · US

Funding

NHLBI NIH HHS K99 HL130035NHLBI NIH HHS R00 HL130035NHLBI NIH HHS R01 HL153054NIAMS NIH HHS L40 AR068727NICHD NIH HHS P50 HD090254NICHD NIH HHS U54 HD090257
6 · The paper itself

Abstract

There is no approved therapy for Becker muscular dystrophy (BMD), a genetic muscle disease caused by in-frame dystrophin deletions. We previously developed the dissociative corticosteroid vamorolone for treatment of the allelic, dystrophin-null disease Duchenne muscular dystrophy. We hypothesize vamorolone can treat BMD by safely reducing inflammatory signaling in muscle and through a novel mechanism of increasing dystrophin protein via suppression of dystrophin-targeting miRNAs. Here, we test this in the

Indexed as

Biological sciencesNeurosciencePharmacology

Identifiers

PMID37534133
PMCPMC10391915
OpenAlexW4380987570

What Socratic holds

Textmetadata
LicenceCC BY
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 Socratic graph.