Blood Biomarkers Help Assess Growth Risks During Steroid Treatment in Duchenne Muscular Dystrophy
Posted on 09 Sep 2026
Children with Duchenne muscular dystrophy (DMD) commonly receive long-term corticosteroids to slow disease progression, but these drugs can impair linear growth and weaken bone. Understanding how specific treatments affect skeletal biology at the growth plate remains a clinical need, as does identifying minimally invasive ways to monitor these effects during therapy. A new study shows that serum biomarker patterns can distinguish prednisone from vamorolone and may help explain treatment-related growth complications.
Binghamton University (Binghamton, NY, USA) researchers and international collaborators identified a panel of circulating proteins that function as bone and cartilage biomarkers during corticosteroid therapy in young boys with DMD. The work delineates a prednisone-specific signature that aligns with suppression of growth-plate activity and bone turnover, while indicating an absence of this effect with vamorolone, a drug created by a Binghamton researcher and used to treat DMD. The findings, published in Scientific Reports on August 3, 2026, suggest a blood-based readout of skeletal biology that could complement clinical assessments.
The team first evaluated four commonly used clinical laboratory markers of bone formation and turnover—alkaline phosphatase (ALP), osteocalcin, procollagen type I N‑terminal propeptide (P1NP), and collagen type I C‑terminal telopeptide (CTX1). All four declined during prednisone treatment but remained stable with vamorolone or placebo, and values recovered after prednisone was tapered and participants transitioned to vamorolone. A broader protein screen then identified 10 additional bone- and cartilage-related proteins reduced by prednisone; many are associated with osteoblasts and specialized growth‑plate cells.
The analysis draws on a double-blind clinical trial in boys aged 4 to 7 years. During the first 24 weeks, participants received placebo, prednisone at 0.75 mg/kg/day, or vamorolone at 6 mg/kg/day. Over the next 24 weeks, those initially assigned to placebo or prednisone crossed over to vamorolone, while the original vamorolone group continued treatment. Investigators emphasize that the biomarker analysis involved a smaller subset of the overall trial and that blood biomarkers do not necessarily correspond directly to clinical outcomes.
According to the authors, the prednisone-associated biomarker pattern was not seen with vamorolone or placebo, and suppressed markers returned toward pretreatment levels after patients switched from prednisone to vamorolone. The study notes that larger and longer studies are needed to determine whether these biomarkers predict later skeletal outcomes and how they perform in girls or in other pediatric conditions. Collaborators included Carleton University as part of an international, multidisciplinary team.
“Prednisone remains an important therapy for Duchenne muscular dystrophy, but its effects on growth and bone health are a major concern for children and families. Importantly, both prednisone and vamorolone improved motor outcomes in the trial, but only prednisone produced this adverse biomarker pattern,” said Eric P. Hoffman, senior author of the study and associate dean for research and research development and professor of pharmaceutical sciences at Binghamton University.
“Following the same participants through the treatment crossover was particularly informative. The prednisone-associated changes appeared in both standard clinical laboratory tests and the broader analysis, and the markers moved back toward baseline after prednisone was discontinued. That pattern supports a reversible drug effect rather than a change caused only by progression of Duchenne muscular dystrophy,” said Utkarsh Dang, lead statistician of the study and a professor at Carleton University’s Department of Health Sciences.
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