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New blood biomarkers reveal how common muscular dystrophy treatment may impair overall growth

In a study published in Scientific Reports, an international, multidisciplinary research team that included Binghamton University researchers identified a set of blood proteins that may explain why the corticosteroid prednisone, although beneficial in treating Duchenne muscular dystrophy (DMD), can interfere with childhood growth and bone health.

New blood biomarkers reveal how common muscular dystrophy treatment may impair overall growth

An international research team, including scientists from Binghamton University, identified specific blood proteins that may explain why the corticosteroid prednisone, used to treat Duchenne muscular dystrophy (DMD), can negatively impact growth and bone health in children. The findings emerged from a clinical trial with boys aged 4-7 with DMD who received different treatments over 48 weeks.

Prednisone significantly decreased markers of bone formation, turnover, and growth-plate activity, while vamorolone or placebo did not. When children switched from prednisone to vamorolone, the suppressed biomarkers returned to their original levels. Prednisone and vamorolone both improved motor outcomes in the trial, but only prednisone caused the adverse biomarker pattern.

The researchers found that prednisone suppressed four common clinical markers of bone formation and identified 10 additional proteins reduced by prednisone. Many of these proteins are linked to osteoblasts, which form bone, or specialized cells in the growth plate that aid in bone lengthening. The study suggests prednisone may be reversible and may help monitor the biological health of the growth plate during treatment for DMD and potentially other pediatric conditions.

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