Abstract #0566

Monitoring glycogen levels and treatment effect in a mouse model of Pompe disease

Qing Zeng^1,2, Yuguo Li^1,2, Derek Timm³, Tyler Johnson³, Nickita Mehta³, Brian Fox³, Peter C. M. van Zijl^1,2, and Nirbhay N. Yadav^1,2

¹Russell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins University School of Medicine, Baltimore, MD, United States, ²F.M. Kirby Research Center for Functional Brain Imaging, Kennedy Krieger Institute, Baltimore, MD, United States, ³Amicus Therapeutics, Inc., Princeton, NJ, United States

Synopsis

Keywords: Muscle, Rare disease, metabolism, molecular imaging

Motivation: Pompe disease is a glycogen storage disease featuring abnormal glycogen accumulation in tissues. Noninvasive methods for assessing disease progression and treatment effect are highly needed.

Goal(s): To use glycoNOE MRI to assess muscle glycogen levels in Pompe disease and the effect of treatment on these levels.

Approach: GlycoNOE MRI and a lineshape fitting model were used to detect glycogen levels in skeletal muscle in a mouse model of Pompe disease. Various therapies were assessed.

Results: GlycoNOE MRI glycogen quantification can discriminate between controls, mice with Pompe disease, and different treatment effects.

Impact: Monitoring of glycogen levels is essential for assessing Pompe disease load and treatment efficacy. GlycoNOE MRI offers the possibility to non-invasively map glycogen levels in tissue using standard MRI scanners.

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