Evaluation of a serum protein signature as monitoring biomarker for Duchenne muscular dystrophy in a long-term clinical trial with corticosteroids.

What was studied

This study asked if blood proteins can track function in Duchenne muscular dystrophy. It used samples from the FOR-DMD trial. That trial compared daily and intermittent steroid plans in boys aged 4-8 years. A lab platform measured 1500 proteins. Models linked protein levels to tests of movement and walking.

What they found

Some proteins, including RGMA, ART3, ANTXR2 and CFB, linked to scores on several function tests. This held across boys and within each boy over time. Models that used the proteins predicted scores better than a simple model. The simple model used only age and treatment. A set of proteins was picked again and again by the models. Lab readings of CFB, RGMA, ANTXR2, SERPINF1 and ATP5PF agreed well with other methods.

What it means for patients

The authors say serum protein patterns can act as objective measures. They say these track DMD progression and treatment response. They say this could work at clinic visits and in trials.

Why it is not certain yet

The abstract does not say if the signature works outside this trial. The boys all came from one trial. A link between proteins and function does not show cause. The abstract does not show that the tool helps patients.

Source

Evaluation of a serum protein signature as monitoring biomarker for Duchenne muscular dystrophy in a long-term clinical trial with corticosteroids.

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