This is a natural history study for children up to 18 years of age who have been diagnosed with Mucopolysaccharidosis Type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B). Mucopolysaccharidosis type IIIB is a severe neurodegenerative disorder. The information gathered from this trial may help inform the design and interpretation of subsequent interventional studies. No clinical intervention or study drug is provided by Allievex in this study.
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Neurocognitive function
Timeframe: Baseline + every 24 weeks for up to 240 weeks
Behavioral function
Timeframe: Baseline + every 24 weeks for up to 240 weeks
Quality of Life Tests
Timeframe: Baseline + every 24 weeks for up to 240 weeks
Sleep habits
Timeframe: Baseline + every 24 weeks for up to 240 weeks
Disease-specific Biomarkers
Timeframe: Baseline + every 24 weeks for up to 240 weeks
Biochemical, Molecular, Cellular and Genetic Markers of Disease Burden
Timeframe: Once (at baseline visit)