Abstract #5130

Phosphodiester-levels in Muscle Assessed using 31P MRS are an Early Marker for Disease Activity in DMD

Melissa T. Hooijmans¹, Nathalie Doorenweerd^1,2, Jedrzej Burakiewicz¹, Celine A. Baligand¹, Jan J.G.M. Verschuuren², Andrew G. Webb¹, Erik H. Niks², and Hermien E. Kan¹

¹C.J.Gorter Center for High-field MRI, Dept. of Radiology, Leiden University Medical Center, Leiden, Netherlands, ²Dept. of Neurology, Leiden University Medical Center, Leiden, Netherlands

Quantitative MR of muscle is increasingly important as potential outcome measure for therapy development in DMD. Since therapy is aimed at preserving or improving muscle tissue, an early marker that reflects muscle state with a suitable dynamic range is essential. Unfortunately, water T2 and %fat do not meet this criteria. Therefore, we aimed to assess whether phosphodiester (PDE)-levels detected by 31P MRS could fill this gap. We have shown a two-fold increase in PDE-levels compared to controls and its detection prior to structural changes which confirm the potential of PDE as an early marker for disease activity in DMD patients.

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