Evidence map›Paper›PMID 39715964›Full record

SynthesisNeurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology2025

Meta-analysis of the efficacy and safety of vamorolone in Duchenne muscular dystrophy.

Mohamed Said Ibrahim, Omar Ahmed Abdelwahab, Bashaer Elawfi, Fatmaelzahraa Yasser Ali, Sarah Amro, Shrouk F Mohammed, Nour Shaheen, Ahmed Negida, Martin Arndt, Mido Max Hijazi and 2 more

Abstract readMeta-AnalysisSystematic Review
PubMed Full text at PMC Publisher
In one paragraph

Synthesis in Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed, 1 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.

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

4 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
  3. Article
  4. Polypharmacology: new drugs in 2023-2024.Pharmacological reports : PR · 2025
    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

12 authors.

Mohamed Said IbrahimDresden International University, Division of Medicine, Dresden, Germany.
Omar Ahmed AbdelwahabMedical Research Group of Egypt, Negida Academy, Arlington, MA, USA.
Bashaer ElawfiMedical Research Group of Egypt, Negida Academy, Arlington, MA, USA.
Fatmaelzahraa Yasser AliMedical Research Group of Egypt, Negida Academy, Arlington, MA, USA.
Sarah AmroMedical Research Group of Egypt, Negida Academy, Arlington, MA, USA.
Shrouk F MohammedMedical Research Group of Egypt, Negida Academy, Arlington, MA, USA.
Nour ShaheenMedical Research Group of Egypt, Negida Academy, Arlington, MA, USA.
Ahmed NegidaMedical Research Group of Egypt, Negida Academy, Arlington, MA, USA.
Martin ArndtDepartment of Neurology, Medical Faculty and University Hospital Carl Gustav Carus, TUD Dresden University of Technology, Dresden, Germany.
Mido Max HijaziDepartment of Neurosurgery, Medical Faculty and University Hospital Carl Gustav Carus, TUD Dresden University of Technology, Dresden, Germany.
Jochen SchaeferDepartment of Neurology, Medical Faculty and University Hospital Carl Gustav Carus, TUD Dresden University of Technology, Dresden, Germany.
Timo SiepmannDresden International University, Division of Medicine, Dresden, Germany. timo.siepmann@ukdd.de.ORCID http://orcid.org/0000-0003-2196-5904

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundDuchenne muscular dystrophy (DMD) is a severe neuromuscular disorder, often leading to wheelchair dependence by age 13 with limited treatment options, largely relying on glucocorticosteroids. We assessed the efficacy and safety of vamorolone, a modified synthetic corticosteroid, for DMD.

methodsWe performed a systematic review and meta-analysis using seven databases including prospective studies comparing vamorolone with glucocorticosteroids or placebo in DMD patients. We extracted data on efficacy and safety outcomes. We built fixed effects models to assess mean differences. (PROSPERO: CRD42023396908).

resultsOut of 210 identified records, two study reports were included in meta-analysis providing data from 210 patients. Vamorolone at 2.0 mg/kg/day was associated with improvement time to climb four stairs velocity (MD = 0.05 95% CI [0.03 to 0.08] P = 0.0002), and time stand from supine velocity (MD = 0.07 95% CI [0.01 to 0.07] P = 0.007). A higher dose of 6.0 mg/kg/day was additionally associated with higher time to run/walk 10 m velocity (MD = 0.10 95% CI [-0.0.1 to 0.21] P = 0.07, I2 = 0%). Among these beneficial effects only improvement in time to climb four stairs velocity was sustained after a follow-up period of 30 months. Vamorolone did not inhibit growth but increased the risk of weight gain, suppression of adrenal function, and insulin resistance.

conclusionThe results of our systematic review and meta-analyis are suggestive of improved efficacy and safety of vamorolone for DMD compared to standard glucocorticosteroids but the external validity of these findings as well as the medication's long-term effects remain to be determined.

Indexed as

Muscular Dystrophy, DuchenneHumansPregnadienediolsTreatment OutcomePregnadienediolsVBP15 compound6MWTDrugMuscleNSAASteroidTTCLIMBTTRWTTSTANDVamorolone

Identifiers

PMID39715964
PMCPMC12003609

What OpenQuestion holds

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Registered trials

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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.