Muscle function, linear growth, and bone health during 30 months of vamorolone, prednisone, and deflazacort treatment for Duchenne muscular dystrophy: a comparative study

Clicks: 8
ID: 337676
2026
Free PDF
Article Quality & Performance Metrics
Overall Quality
Not rated
Combines reader engagement with the AI quality analysis. This article has not been analysed, so there is no overall score — reader engagement is measured and shown alongside.
AI Quality Assessment
Not analyzed
Readership in this journal
Emerging

Ranked #142 of 143 articles by views in journal of bone and mineral research : the official journal of the american society for bone and mineral research

Most read Least read

Bar heights use a square-root scale. Only the 120 most-read articles are drawn; the journal has 143 in total.

Mint this article as an NFT
Not yet minted

Create a permanent, verifiable on-chain record of this article on the Scimatic Network. The NFT is held in your Journament account, and you can withdraw it to your own wallet at any time.

5 SUSD one-off · no wallet required
Sign in to mint
Abstract
Abstract Corticosteroids are standard of care to slow disease progression in Duchenne muscular dystrophy (DMD) despite impaired growth and increased fracture risk. Vamorolone, a dissociative corticosteroid approved for DMD, showed stable growth and serum bone turnover markers in the 48-week VISION-DMD study. We compared muscle function and bone health from the non-randomized, open-label, 30-month Phase 2a VBP15-002/003/LTE trials (NCT02760264/NCT02760277/NCT03038399) in treatment-naive boys with DMD, aged 4 to <7 years at baseline, with data from matched corticosteroids-treated boys from FOR-DMD (NCT01603407). Motor function by slope-of-change analysis was evaluated for vamorolone 2–6 mg/kg/d through 30 months compared with propensity score-matched boys from FOR-DMD who received daily prednisone (starting dosage 0.75 mg/kg/d) or deflazacort (0.9 mg/kg/d) for 36 months. Anthropometric and bone assessments were performed in boys on vamorolone for 30 months, including those who received lower dosages during VBP15-002/003 (0.25/0.75 mg/kg/d for 6 months). Vertebral fracture (VF) prevalence was compared with baseline-matched FOR-DMD data. Thirty-month vamorolone VF results were adjusted to approximate expected fracture prevalence at 36 months. All slope-of-change motor function comparisons between vamorolone (N=22) and prednisone (N=32) or deflazacort (N=34) groups yielded no significant differences (p>0.05). Thirty-month height z-scores remained stable from baseline for vamorolone (N=36; +0.078 least-squares-mean change) and decreased from baseline for prednisone (N=27, –0.668; change versus vamorolone: p<0.0001 mixed models for repeat measures) and deflazacort (N=28, –0.928; change versus vamorolone: p<0.0001). VF prevalence on vamorolone (8 VF in 5/39 boys [12.8%]) was ~50% lower than prednisone (13 VF in 7/27 boys [25.9%]) and deflazacort (19 VF in 8/28 boys [28.6%]). Compared with prednisone and deflazacort, vamorolone demonstrated similar motor function efficacy with significantly fewer VF and preserved growth.
Reference Key
openalex_W7224225180 Use this key to autocite in the manuscript while using SciMatic Manuscript Manager or Thesis Manager
Authors Leanne Marie Ward, Stefan A. Jackowski, Utkarsh J. Dang, Maya Scharke B Comm, Jacob Lester Jaremko, Khaldoun Koujok, Mary‐Ann Matzinger, Nazih F. Shenouda, Hugh J. McMillan, Kerry G. Siminoski, Mika Leinonen, Raoul Rooman, Shabir Hasham, Paula R. Clemens, Michela Guglieri, Eric P. Hoffman
Are you one of these authors? Claim your profile
Journal journal of bone and mineral research : the official journal of the american society for bone and mineral research
Year 2026
DOI
10.1093/jbmr/zjag154
URL
https://doi.org/10.1093/jbmr/zjag154
Keywords Keywords not found

Citations

No citations found. To add a citation, contact the admin at info@scimatic.org

Comments

No comments yet. Be the first to comment on this article.