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

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DataCite Commons2025-06-01 更新2026-05-07 收录
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https://search.vivli.org/doiLanding/studies/PR00010330/isLanding
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资源简介:
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.
提供机构:
Vivli
创建时间:
2024-07-18
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