Grace Oberle

Past research has found several possible magnetic resonance (MR) biomarkers for disease progression of Mucopolysaccharidosis (MPS) IIIA, but their validity is limited by only tracking subjects for one to two years. The objectives of this study were to build upon the biomarkers proposed by Shapiroe et al. (2016), and identify other potential indicators of MPS IIIA disease progression by analyzing brain MR data of MPS IIIA subjects collected over four years. MR data collected as part of a Takeda MPS IIIA clinical trial (https://proxy.qualtrics.com/proxy/?url=https%3A%2F%2Fwww.clinicaltrials.gov%2Fct2%2Fshow%2FNCT01299727&token=4Hl5wQhGl9z6z2Fo2jrQ4QpjTlffLxev7TPuryrXnUg%3D) was used for analysis. At the start, twelve subjects aged 4 to 25 were scanned using MRI over a course of four years. The volumetric analysis software Freesurfer was used to automatically segment the brain to quantify the volumes of gray/white matter, ventricles, and other structures. Manual interventions were performed by the researcher to fix incorrectly segmented boundaries. The obtained brain volumes, including cortical gray matter, cortical white matter, ventricle size, etc. were analyzed for trends over time. Results showed that as time from the initial scan increased, the listed variables above showed a slight decrease in volume. Excluding ventricle size, these findings align with the results from Shapiro et al. (2016). The results of this study provide further evidence on the validity and possible usefulness of potential MR biomarkers for the progression of MPS IIIA.