Disease progression biomarkers in Duchenne muscular dystrophy

Takeaway

  • Disease progression and muscle involvement in those with Duchenne muscular dystrophy (DMD) was highly variable.

  • Modeling changes in the biomarkers magnetic resonance spectroscopy (MRS) fat fraction (FF) and MRI quantitative T2 (qT2) can be used to evaluate the impact of corticosteroids on disease progression, and are sensitive, noninvasive, measures.

Why this matters

  • There is no cure currently available for DMD, but treatments have been identified and undergoing clinical evaluation.

  • For more rapid evaluation of these exciting treatment possibilities, improved knowledge of the biomarkers relating to disease progression is greatly sought.