A Longitudinal, Prospective, Natural History Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
收藏DataCite Commons2025-06-01 更新2026-05-07 收录
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https://search.vivli.org/doiLanding/studies/PR00010325/isLanding
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资源简介：
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.
提供机构：
Vivli
创建时间：
2024-07-18
