The purpose of the study is to characterize structural abnormalities in the brain and the integrity of the blood brain barrier in patients with mucopolysaccharidosis type IIIB (MPS IIIB).
Study Type
OBSERVATIONAL
Enrollment
5
Unnamed facility
Birmingham, United Kingdom
Blood Brain Barrier integrity in MPS IIIB subjects
Blood Brain Barrier integrity in MPS IIIB subjects by estimating the CSF-AI.
Time frame: Day 0
Blood Brain Barrier transfer coefficient
The Blood Brain Barrier transfer coefficient will be measured by DCE-MRI in MPS IIIB subjects.
Time frame: Day 0
Structural brain abnormalities in MPS IIIB
Structural brain abnormalities in MPS IIIB using imaging and biomarkers related to underlying disease biology of MPS IIIB subjects
Time frame: Day 0
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