Dataset from A Longitudinal, Prospective, Natural History Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)

The purpose is to evaluate the course of disease progression in MPS IIIA patients who are untreated to identify potential surrogate endpoints that may be utilized in future ERT trials of MPS IIIA via defined assessments including standardized clinical, biochemical, neurocognitive, behavioral, developmental, and imaging measures.

本数据集旨在评估未接受治疗的黏多糖贮积症IIIA型（MPS IIIA）患者的疾病进展进程，通过涵盖标准化临床、生化、神经认知、行为、发育及影像检测的既定评估手段，识别可用于未来黏多糖贮积症IIIA型酶替代疗法（Enzyme Replacement Therapy, ERT）临床试验的潜在替代终点。