Microstructural and functional MRI signatures in patients with MPS I

PI: Igor Nestrasil, MD

Institution: University of Minnesota

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Mucopolysaccharidosis type I (MPS I) is considered to be primarily caused by altered myelination, but the disease still lacks comprehensive prognostic markers to track progressive brain changes and responses to newly developed treatments. In this project we utilized a novel non-invasive magnetic resonance imaging (MRI) technique recently developed by our group, entitled Relaxation Along a Fictitious Field in the rotating frame of rank n (RAFFn). RAFFn was shown to be highly sensitive to myelin content and integrity in murine model of MPSI. We assume that the assessment of brain disease with the use of highly sensitive MRI parameters holds incredible potential to establish disease-specific biomarkers. These measures may be employed in clinical trials and will help to determine the most efficient treatment of central nervous disease in MPS I.