Dataset from An Observational Prospective Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

The purpose of this study is to evaluate the natural course of disease progression in Mucopolysaccharidosis Type III (MPS IIIB) patients who are untreated to identify potential surrogate endpoints that may be utilized in future treatment trials of MPS IIIB using predefined assessments including standardized clinical, biochemical, neurocognitive, developmental, and imaging measures.

本研究旨在评估未接受治疗的黏多糖贮积症Ⅲ型B型（Mucopolysaccharidosis Type III, MPS IIIB）患者的疾病自然进展过程，并通过预设的评估工具（包括标准化临床、生化、神经认知、发育及影像学评估手段），识别可用于未来MPS IIIB治疗试验的潜在替代终点。